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Archive for the ‘Cell Biology, Signaling & Cell Circuits’ Category

Accelerating PROTAC drug discovery: Establishing a relationship between ubiquitination and target protein degradation

Curator: Stephen J. Williams, Ph.D.

PROTACs have been explored in multiple disease fields with focus on only few ligases like cereblon (CRBN), Von Hippel-Lindau (VHL), IAP and MDM2. Cancer targets like androgen receptor, estrogen receptor, BTK, BCL2, CDK8 and c-MET [[6], [7], [8], [9], [10], [11]] have been successfully targeted using PROTACs. A variety of BET family (BRD2, BRD3, and BRD4)- PROTACs were designed using multiple ligases; MDM2-based BRD4 PROTAC [12], CRBN based dBET1 [13] and BETd-24-6 [14] for triple-negative breast cancer, enhanced membrane permeable dBET6 [15], and dBET57 PROTAC [16]. PROTACs for Hepatitis c virus (HCV) protease, IRAK4 and Tau [[17], [18], [19]] have been explored for viral, immune and neurodegenerative diseases, respectively. Currently, the PROTAC field expansion to vast undruggable proteome is hindered due to narrow focus on select E3 ligases. Lack of reliable tools to rapidly evaluate PROTACs based on new ligases is hindering the progress. Screening platforms designed must be physiologically relevant and represent true PROTAC cellular function, i.e., PROTAC-mediated target ubiquitination and degradation.

In the current study, we employ TUBEs as affinity capture reagents to monitor PROTAC-induced poly-ubiquitination and degradation as a measure of potency. We established and validated proof-of-concept cell-based assays in a 96-well format using PROTACS for three therapeutic targets BET family proteins, kinases, and KRAS. To our knowledge, the proposed PROTAC assays are first of its kind that can simultaneously 1) detect ubiquitination of endogenous, native protein targets, 2) evaluate the potency of PROTACs, and 3) establish a link between the UPS and protein degradation. Using these TUBE assays, we established rank order potencies between four BET family PROTACs dBET1, dBET6, BETd246 and dBET57 based on peak ubiquitination signals (“UbMax”) of the target protein. TUBE assay was successful in demonstrating promiscuous kinase PROTACs efficiency to degrade Aurora Kinase A at sub-nanomolar concentrations within 1 h. A comparative study to identify changes in the ubiquitination and degradation profile of KRAS G12C PROTACs recruiting two E3 ligases (CRBN and VHL). All of the ubiquitination and degradation profiles obtained from TUBE based assays correlate well with traditional low throughput immunoblotting. Significant correlation between DC50 obtained from protein degradation in western blotting and UbMax values demonstrates our proposed assays can aid in high-throughput screening and drastically eliminate artifacts to overcome bottlenecks in PROTAC drug discovery.

To successfully set up HTS screening with novel PROTACs without pre-existing knowledge, we recommend the following steps. 1. Identify a model PROTAC that can potentially demonstrate activity based on knowledge in PROTAC design or in vitro binding studies. 2. Perform a time course study with 2–3 doses of the model PROTAC based on affinities of the ligands selected. 3. Monitor ubiquitination and degradation profiles using plate-based assay and identify time point that demonstrates UbMax. 4. Perform a dose response at selected time point with a library of PROTACs to establish rank order potency.

INTRODUCTION

Ubiquitination is a major regulatory mechanism to maintain cellular protein homeostasis by marking proteins for proteasomal-mediated degradation [1]. Given ubiquitin’s role in a variety of pathologies, the idea of targeting the Ubiquitin Proteasome System (UPS) is at the forefront of drug discovery [2]. “Event-driven” protein degradation using the cell’s own UPS is a promising technology for addressing the “undruggable” proteome [3]. Targeted protein degradation (TPD) has emerged as a new paradigm and promising therapeutic option to selectively attack previously intractable drug targets using PROteolytic TArgeting Chimeras (PROTACs) [4]. PROTACs are heterobifunctional molecules with a distinct ligand that targets a specific E3 ligase which is tethered to another ligand specific for the target protein using an optimized chemical linker. A functional PROTAC induces a ternary E3-PROTAC-target complex, resulting in poly-ubiquitination and subsequent controlled protein degradation [5]. Ability to function at sub-stoichiometric levels for efficient degradation, a significant advantage over traditional small molecules.

PROTACs have been explored in multiple disease fields with focus on only few ligases like cereblon (CRBN), Von Hippel-Lindau (VHL), IAP and MDM2. Cancer targets like androgen receptorestrogen receptor, BTK, BCL2, CDK8 and c-MET [[6][7][8][9][10][11]] have been successfully targeted using PROTACs. A variety of BET family (BRD2, BRD3, and BRD4)- PROTACs were designed using multiple ligases; MDM2-based BRD4 PROTAC [12], CRBN based dBET1 [13] and BETd-24-6 [14] for triple-negative breast cancer, enhanced membrane permeable dBET6 [15], and dBET57 PROTAC [16]. PROTACs for Hepatitis c virus (HCV) proteaseIRAK4 and Tau [[17][18][19]] have been explored for viral, immune and neurodegenerative diseases, respectively. Currently, the PROTAC field expansion to vast undruggable proteome is hindered due to narrow focus on select E3 ligases. Lack of reliable tools to rapidly evaluate PROTACs based on new ligases is hindering the progress. Screening platforms designed must be physiologically relevant and represent true PROTAC cellular function, i.e., PROTAC-mediated target ubiquitination and degradation.

Cellular PROTAC screening is traditionally performed using cell lines harboring reporter genes and/or Western blotting. While Western blotting is easy to perform, they are low throughput, semi-quantitative and lack sensitivity. While reporter gene assays address some of the issues, they are challenged by reporter tags having internal lysines leading to artifacts. Currently, no approaches are available that can identify true PROTAC effects such as target ubiquitination and proteasome-mediated degradation simultaneously. High affinity ubiquitin capture reagents like TUBEs [20] (tandem ubiquitin binding entities), are engineered ubiquitin binding domains (UBDs) that allow for detection of ultralow levels of polyubiquitinated proteins under native conditions with affinities as low as 1 nM. The versatility and selectivity of TUBEs makes them superior to antibodies, and they also offer chain-selectivity (-K48, -K63, or linear) [21]. High throughput assays that can report the efficacy of multiple PROTACs simultaneously by monitoring PROTAC mediated ubiquitination can help establish rank order potency and guide chemists in developing meaningful structure activity relationships (SAR) rapidly.

In the current study, we employ TUBEs as affinity capture reagents to monitor PROTAC-induced poly-ubiquitination and degradation as a measure of potency. We established and validated proof-of-concept cell-based assays in a 96-well format using PROTACS for three therapeutic targets BET family proteins, kinases, and KRAS. To our knowledge, the proposed PROTAC assays are first of its kind that can simultaneously 1) detect ubiquitination of endogenous, native protein targets, 2) evaluate the potency of PROTACs, and 3) establish a link between the UPS and protein degradation. Using these TUBE assays, we established rank order potencies between four BET family PROTACs dBET1, dBET6, BETd246 and dBET57 based on peak ubiquitination signals (“UbMax”) of the target protein. TUBE assay was successful in demonstrating promiscuous kinase PROTACs efficiency to degrade Aurora Kinase A at sub-nanomolar concentrations within 1 h. A comparative study to identify changes in the ubiquitination and degradation profile of KRAS G12C PROTACs recruiting two E3 ligases (CRBN and VHL). All of the ubiquitination and degradation profiles obtained from TUBE based assays correlate well with traditional low throughput immunoblotting. Significant correlation between DC50 obtained from protein degradation in western blotting and UbMax values demonstrates our proposed assays can aid in high-throughput screening and drastically eliminate artifacts to overcome bottlenecks in PROTAC drug discovery.

Fig. 1. Schematic representation of TUBE assay to monitor PROTAC mediated cellular ubiquitination of target proteins.
Fig. 2. TUBE based assay screening of PROTACs: Jurkat cell lysates were treated with BRD3-specific PROTACs A) dBET1, B) dBET6, C) BETd24-6, and D) dBET57. Polyubiquitination profiles and Ubmax of BRD3 for each PROTAC were represented as relative CL intensity. Relative CL intensities were calculated by dividing raw CL signals from a given PROTAC dose over DMSO treated samples. Error bars represent standard deviations, n = 3.
Fig. 3. PROTAC mediated degradation of bromodomain proteins analyzed by anti-BRD3 western blotting. Dose response of PROTACs dBET1, dBET6, Betd-24-6 and dBET57 at 45 min in Jurkat cells demonstrates degradation of BRD3, Acting as loading control.

 

 

 

 

 

 

 

 

 

Fig. 4. PROTAC mediated ubiquitination and degradation of AURKA in K562 cells. (A) Time course study to evaluate intracellular ubiquitination and degradation. (B) Western blot analysis of time course study: degradation kinetics (C) A dose response study to evaluate DC50 of the promiscuous kinase PROTAC in K562 cells. (D) Western blot analysis of dose response study to monitor degradation, GAPDH as loading control. Error bars represent standard deviation, n = 3.

SOURCE

https://www.sciencedirect.com/science/article/abs/pii/S0006291X22011792

Other articles of PROTACs in this Open Access Journal Include

The Vibrant Philly Biotech Scene: Proteovant Therapeutics Using Artificial Intelligence and Machine Learning to Develop PROTACs

The Map of human proteins drawn by artificial intelligence and PROTAC (proteolysis targeting chimeras) Technology for Drug Discovery

Live Conference Coverage AACR 2020 in Real Time: Monday June 22, 2020 Late Day Sessions

From High-Throughput Assay to Systems Biology: New Tools for Drug Discovery

 

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2022 Albert Lasker Basic Medical Research Award for Integrins—Mediators of Cell-Matrix and Cell-Cell Adhesion

Reporter: Aviva Lev-Ari, PhD, RN

 

The three recipients of 2022 Albert Lasker Basic Medical Research Award For discoveries concerning the integrins—key mediators of cell-matrix and cell-cell adhesion in physiology and disease are:

  • Richard O. Hynes, Massachusetts Institute of Technology
  • Erkki Ruoslahti, Sanford Burnham Prebys
  • Timothy A. Springer, Boston Children’s Hospital/Harvard Medical School

2022 Basic Award video

https://www.youtube.com/watch?v=4k60P3Pnh30

Acceptance remarks

Watch 3 Videos
 
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Figure 1A: Pairs that bond
Two protein chains, α and ß, compose the heterodimeric integrins that span the cell membrane. The portion outside affixes to molecules in the extracellular matrix (ECM) or on other cells. Adherence through integrins triggers cytoskeleton assembly and vice versa. Such events can also influence the behavior of other proteins inside the cell. The image shows the matrix-cell type of integrin; a second type mediates cell-cell interactions.
Illustration: Cassio Lynm / © Amino Creative

Figure 1B: Families that stick together
Integrin α and ß subunit each belong to a family of proteins with a shared evolutionary ancestor. Individual members of the α and ß families combine in different ways to create 24 mammalian heterodimers that physically associate with a variety of molecules. In the ECM, these binding partners include collagen, laminin, and other RGD-containing proteins such as fibronectin and fibrinogen; on cells, these partners include proteins such as ICAM-1, which dwells on the surface of white blood cells and cells that line blood vessels. The first integrin identified, the platelet receptor, promotes blood clotting. LFA-1 helps immune cells fight infections in numerous ways.
Illustration: Cassio Lynm / © Amino Creative

Figure 2: Fleeting attachments, profound effects
By fostering adherence between cells and the ECM or other cells, integrins play a key role in a tremendous number of essential physiological interactions.
Illustration: Cassio Lynm / © Amino Creative

Figures Source: 

https://laskerfoundation.org/winners/integrins-mediators-of-cell-matrix-cell-adhesion/

 

Selected Publications – Discovery of Integrins

Cell-Matrix Interactions

Ruoslahti, E., Vaheri. A., Kuusela, P., and Linder, E. (1973). Fibroblast surface antigen: a new serum protein. Biochim. BiophysActa. 322, 352-358.

Hynes, R.O. (1973). Alteration of cell-surface proteins by viral transformation and by proteolysis. Proc. Natl. Acad. Sci. USA70, 3170-3174.

Pierschbacher, M.D., and Ruoslahti, E. (1984). Cell attachment activity of fibronectin can be duplicated by small synthetic fragments of the molecule. Nature309, 30-33.

Pytela, R., Pierschbacher, M.D., and Ruoslahti, E. (1985). A 125/115-kDa cell surface receptor specific for vitronectin interacts with the arginine-glycine-aspartic acid adhesion sequence derived from fibronectin. Proc. Natl. Acad. Sci. USA. 82, 5766-5770.

Pytela, R., Pierschbacher, M.D., Ginsberg, M.H., Plow, E.F., and Ruoslahti, E. (1986). Platelet membrane glycoprotein IIb/IIIa: member of a family of Arg-Gly-Asp-specific adhesion receptors. Science231, 1559-1562.

Tamkun, J.W., DeSimone, D.W., Fonda, D., Patel, R.S., Buck, C., Horwitz, A.F., and Hynes, R.O. (1986). Structure of integrin, a glycoprotein involved in the transmembrane linkage between fibronectin and actin. Cell. 46, 271-282.

Hynes, R.O. (1987). Integrins: a family of cell surface receptors. Cell48, 549-554.

Ruoslahti, E. (1996). RGD and other recognition sequences for integrins. Annu. Rev. Cell Dev. Biol. 12, 697-715.

Hynes, R.O. (2002). Integrins: bidirectional allosteric signaling machines. Cell. 110, 673-687.

Cell-Cell Interactions

Kurzinger, K., Reynolds, T., Germain, R.N., Davignon, D., Martz, E., and Springer, T.A. (1981). A novel lymphocyte function-associated antigen (LFA-1): cellular distribution, quantitative expression, and structure. J. Immunol. 127, 596-600.

Sanchez-Madrid, F., Nagy, J., Robbins, E., Simon, P., and Springer, T.A. (1983). A human leukocyte differentiation antigen family with distinct alpha subunits and a common beta subunit: the lymphocyte function-associated antigen (LFA-1), the C3bi complement receptor (OKM1/Mac-1), and the p150,95 molecule. J. Exp. Med. 158, 1785-1789.

Thompson, W.S., Miller, L.J., Schmalstieg, F.C., Anderson, D.C., and Springer, T.A. (1984). Inherited deficiency of the Mac-1, LFA-1, p150,95 glycoprotein family and its molecular basis. J. Exp. Med. 160, 1901-1905.

Kishimoto, T.K., Lee, A., Roberts, T.M., and Springer, T.A. (1987). Cloning of the beta subunit of the leukocyte adhesion proteins: homology to an extra-cellular matrix receptor defines a novel supergene family. Cell. 48, 681-690.

Makgoba. M.W., Sanders, M.E., Luce, G.E.G., Dustin, M.L., Springer, T.A., Clark, E.A., Mannoni, P., and Shaw, S. (1988). ICAM-1 a ligand for LFA-1-dependent adhesion of B, T and myeloid cells. Nature331, 86-88.

Staunton, D.E., Dustin, M.L., and Springer, T.A. (1989). Functional cloning of ICAM-2, a cell adhesion ligand for LFA-1 homologous to ICAM-1. Nature. 339, 61-65.

Springer, T.A. (1990). Adhesion receptors of the immune system. Nature. 346, 425-434.

Lawrence, M.B., and Springer, T.A. (1991). Leukocytes roll on a selectin at physiologic flow rates: distinction from and prerequisite for adhesion through integrins. Cell. 65, 859-873.

Luo, B.-H., Carman, C.V., and Springer, T.A. (2007) Structural basis of integrin regulation and signaling. Annu. Rev. Immunol. 25, 619-647.

Li, J., Yan, J., and Springer, T.A. (2021). Low-affinity integrin states have faster ligand-binding kinetics than the high-affinity state. eLife. 10, 1-22. e73359. doi: 10.7554/eLife.73359.

SOURCE

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Reporter and Curator: Dr. Sudipta Saha, Ph.D.

Infertility has been primarily treated as a female predicament but around one-half of infertility cases can be tracked to male factors. Clinically, male infertility is typically determined using measures of semen quality recommended by World Health Organization (WHO). A major limitation, however, is that standard semen analyses are relatively poor predictors of reproductive capacity and success. Despite major advances in understanding the molecular and cellular functions in sperm over the last several decades, semen analyses remain the primary method to assess male fecundity and fertility.

Chronological age is a significant determinant of human fecundity and fertility. The disease burden of infertility is likely to continue to rise as parental age at the time of conception has been steadily increasing. While the emphasis has been on the effects of advanced maternal age on adverse reproductive and offspring health, new evidence suggests that, irrespective of maternal age, higher male age contributes to longer time-to-conception, poor pregnancy outcomes and adverse health of the offspring in later life. The effect of chronological age on the genomic landscape of DNA methylation is profound and likely occurs through the accumulation of maintenance errors of DNA methylation over the lifespan, which have been originally described as epigenetic drift.

In recent years, the strong relation between age and DNA methylation profiles has enabled the development of statistical models to estimate biological age in most somatic tissue via different epigenetic ‘clock’ metrics, such as DNA methylation age and epigenetic age acceleration, which describe the degree to which predicted biological age deviates from chronological age. In turn, these epigenetic clock metrics have emerged as novel biomarkers of a host of phenotypes such as allergy and asthma in children, early menopause, increased incidence of cancer types and cardiovascular-related diseases, frailty and cognitive decline in adults. They also display good predictive ability for cancer, cardiovascular and all-cause mortality.

Epigenetic clock metrics are powerful tools to better understand the aging process in somatic tissue as well as their associations with adverse disease outcomes and mortality. Only a few studies have constructed epigenetic clocks specific to male germ cells and only one study reported that smokers trended toward an increased epigenetic age compared to non-smokers. These results indicate that sperm epigenetic clocks hold promise as a novel biomarker for reproductive health and/or environmental exposures. However, the relation between sperm epigenetic clocks and reproductive outcomes has not been examined.

There is a critical need for new measures of male fecundity for assessing overall reproductive success among couples in the general population. Data shows that sperm epigenetic clocks may fulfill this need as a novel biomarker that predicts pregnancy success among couples not seeking fertility treatment. Such a summary measure of sperm biological age is of clinical importance as it allows couples in the general population to realize their probability of achieving pregnancy during natural intercourse, thereby informing and expediting potential infertility treatment decisions. With the ability to customize high throughput DNA methylation arrays and capture sequencing approaches, the integration of the epigenetic clocks as part of standard clinical care can enhance our understanding of idiopathic infertility and the paternal contribution to reproductive success and offspring health.

References:

https://academic.oup.com/humrep/advance-article/doi/10.1093/humrep/deac084/6583111?login=false

https://pubmed.ncbi.nlm.nih.gov/33317634/

https://clinicalepigeneticsjournal.biomedcentral.com/articles/10.1186/s13148-019-0656-7

https://pubmed.ncbi.nlm.nih.gov/19319879/

https://pubmed.ncbi.nlm.nih.gov/31901222/

https://pubmed.ncbi.nlm.nih.gov/25928123/

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Lessons on the Frontier of Gene & Cell Therapy – The Disruptive Dozen 12 #GCT Breakthroughs that are revolutionizing Healthcare

Reporter: Aviva Lev-Ari, PhD, RN

Mass General Brigham Innovation

@MGBInnovation

Read key takeaways from the 2022 World Medical Innovation Forum in this report from the Bank of America Institute. #WMIF2022

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Bank of America News

@BofA_News

· May 6

What are the 12 emerging #GeneAndCellTherapy technologies with the greatest potential to transform #healthcare? Read our report for key takeaways from #WMIF2022. @MassGenBrigham

4:30 PM · May 6, 2022·Twitter Web App

Mass General Brigham Innovation

@MGBInnovation

Read key takeaways from the 2022 World Medical Innovation Forum in this report from the Bank of America Institute. #WMIF2022

Quote Tweet

Bank of America News

@BofA_News

· May 6

What are the 12 emerging #GeneAndCellTherapy technologies with the greatest potential to transform #healthcare? Read our report for key takeaways from #WMIF2022. @MassGenBrigham

4:30 PM · May 6, 2022·Twitter Web App

The Disruptive Dozen 12 #GCT Breakthroughs that are revolutionizing Healthcare

Liz Everett Krisberg, Head of the Bank of America Institute

The Disruptive Dozen 12 GCT breakthroughs that are revolutionizing healthcare 05 May 2022 Key Takeaways • Gene and cell therapy (GCT) is widely recognized as a transformational opportunity in medicine, with the potential to stop or slow the effects of disease by targeting it at the genetic level. • The “Disruptive Dozen” identifies 12 emerging GCT technologies with the greatest potential to transform healthcare over the next several years • These breakthroughs range from restoration of sight and increasing the supply of donor organs, to treating brain cancer, hearing loss and autoimmune diseases that currently lack few or any treatment alternatives. Gene and cell therapy (GCT) technologies are transforming medicine and the approach to severe diseases like cancer, hereditary conditions including Huntington Disease and Sickle Cell, as well as rare disorders that currently have no treatment alternatives. GCT has the potential to stop or slow the effects of disease by targeting it at the genetic level, either replacing, inactivating or modifying the genetic material or by transferring live or intact cells into a patient to treat or cure disease. Even in cases where the GCT approach does not fully cure a condition, GCT has the potential to be life changing. This is because GCT treatments are often “one and done,” only requiring a single administration, which may enable a patient to manage their disease without onerous ongoing treatment cycles. While some of the first GCT applications were focused on rare and orphan diseases, recent advancements show tremendous potential opportunity for use cases with more broad applications. Beyond the messenger ribonucleic acid or mRNA vaccines that protect against infectious disease including COVID-19, GCT technologies exhibit promise to address prevalent chronic diseases such as diabetes and hearing loss, as well as central nervous system (CNS) disorders and Alzheimer’s. This week, Bank of America joined Mass General Brigham to present the World Medical Innovation Forum in Boston, where over 1,000 clinical experts, industry leaders and investors explored how to advance GCT technologies that may lead to breakthrough medical advancements and solutions. We highlight the twelve emerging GCT technologies – the “Disruptive Dozen” – with the greatest potential to impact and transform healthcare in the next several years. These breakthroughs range from restoration of sight and increasing the supply of donor organs, to treating brain cancer, hearing loss and autoimmune diseases. Restoring sight by mending broken genes Roughly 200 genes are directly linked to vision disorders. In the last several years, groundbreaking new gene therapies have emerged that can compensate for faulty genes in the eye by adding new, healthy copies — a molecular fix that promises to restore sight to those who have lost it. The approach, known as CRISPR-Cas-9 gene editing, could open the door to treating genetic forms of vision loss that are not suited to conventional gene therapy, and a host of other medical conditions. A clinical trial is now underway to evaluate a CRISPR-Cas 9 gene-editing therapy for a severe form of childhood blindness for which there currently are no treatments. Although this treatment is still experimental, it is already historic — it is the first medicine based on CRISPR-Cas-9 to be delivered in vivo, or inside a patient’s body. Similar gene-editing therapies are also under development that correct genes within blood cells. A gene editing solution to increase the supply of donor organs In the U.S. alone, more than 100,000 people need a life-saving organ transplant. But the supply of donor organs is quite limited, and every day, patients die waiting for a donor organ. One way to address this crisis is xenotransplantation — harvesting organs from animals and placing them into human patients. Advances in gene editing technology make it possible to remove, insert, or replace genes with relative ease and precision. This molecular engineering can sidestep the human immune system, which is highly adept at recognizing foreign tissues and triggering rejection. Over the last 20 years, scientists have been working to devise successful gene editing strategies that will render pig organs compatible with humans. The field has taken another major step forward in the past year: transplanting gene-edited pig organs, including the heart and kidney, into humans. While extensive clinical testing is needed before xenotransplantation becomes a reality, that future now seems within reach. I NSTI TUTE Accessible version 2 05 May 2022 I NSTI TUTE Cell therapies to conquer common forms of blindness The eye has been a proving ground for pioneering gene therapies and is also fueling new cell-based therapies than can restore sight, offering a functional cure by replacing critical cells that have been lost or injured. One approach involves stem cells from the retina that can give rise to light-sensitive cells, called photoreceptors, which are required for healthy vision. Scientists are harnessing retinal stem cells to develop treatments for incurable eye diseases, including retinitis pigmentosa. Because the immune system doesn’t patrol the eye as aggressively as other parts of the body, retinal stem cells from unrelated, healthy donors can be transplanted into patients with vision disorders. Other progress includes cell therapies that harness patients’ own cells, for example, from blood or skin, that can be converted into almost any cell type in the body, including retinal cells. Another novel treatment being tested utilizes stem cells from a patient’s healthy eye to repair the affected cornea of the other eye. Harnessing the power of RNA to treat brain cancer RNA is widely known for its helper functions, carrying messages from one part of a cell to another to make proteins. But scientists now recognize that RNA plays a more central role in biology and are tapping its hidden potential to create potent new therapies for a range of diseases, including a devastating form of brain cancer called glioblastoma. This cancer is extremely challenging to treat and highly adaptable. New approaches that either target RNA or mimic its activity could hold promise, including an intriguing class of RNA molecules called microRNAs. One team identified a trio of microRNAs that plays important roles in healthy neurons but is lost when brain cancer develops. These microRNAs can be stitched together into a single unit and delivered into the brain using a virus. Initial studies in mice reveal that this therapeutic can render tumors more vulnerable to existing treatments, including chemotherapy. Another team is also exploring a microRNA called miR-10b. Blocking its activity causes tumor cells to die. Now, scientists are working to develop a targeted therapeutic against miR-10b that can be tested in clinical trials. Realizing the promise of gene therapy for brain disorders Gene therapy holds enormous promise for serious and currently untreatable diseases, including those of the brain and central nervous system. But some big obstacles remain. For example, a commonly-used vehicle for gene therapy — a virus called AAV — cannot penetrate a major biological roadblock, the blood-brain barrier. Now, researchers are engineering new versions of AAV that can cross the blood-brain barrier. Using various molecular strategies, a handful of teams have modified the protein shell that surrounds the virus so it can gain entry and become broadly distributed within the brain. These modified viral vectors are now under development and could begin clinical testing within a few years. Scientists are also tinkering with the inner machinery of AAV to sidestep potential toxicities. With a safe, effective method for accessing the brain, researchers will be able to devise gene therapies for a range of neurological conditions, including neurodegenerative diseases, cancers, and devastating rare diseases that lack any treatment. A flexible, programmable approach to fighting viruses The COVID-19 pandemic has laid bare the tremendous need for rapidly deployable therapies to counteract emerging viruses. Scientists are now developing a novel form of anti-viral therapy that can be programmed to target a range of different viruses — from well-known human pathogens, such as hepatitis C, to those less familiar, such as the novel coronavirus SARS-CoV-2. This new approach harnesses a popular family of gene editing tools, known as CRISPR-Cas. While CRISPR-based systems have gained attention for their capacity to modify human genes, their original purpose in nature was to defend bacteria from viral infections. As a throwback to these early roots, scientists are now adapting CRISPR tools to tackle a variety of viruses that infect humans. Researchers are studying the potential of these programmable anti-viral agents in the context of several different viruses, including ones that pose significant threats to global health, such as SARS-CoV-2, hepatitis C, and HIV. On the move: Cell therapies to restore gut motility The human digestive tract — or “gut” — has its own nervous system. This second brain, known as the enteric nervous system, is comprised of neurons and support cells that carry out critical tasks, like moving food through the gut. When enteric neurons are missing or injured, gut motility can be impaired. Now, scientists are developing an innovative cell replacement therapy to treat diseases of gut motility. Donor cells can be isolated from a patient’s own gut or from a more readily available source, such as subcutaneous fat. These cells are then cultivated in the laboratory and coaxed to form the progenitors that give rise to enteric neurons. Researchers are also devising “off-the-shelf” approaches, which could create a supply of donor cells that are shielded from the immune system and can therefore be transplanted universally across different patients. Early research shows that transplanted enteric neurons can also take up residence in the brain. That means these forays in cell therapy for the gut could also help pave a path toward cell therapies for the brain and spinal cord. CAR-T cell therapies take aim at autoimmune diseases CAR-T cells have emerged as powerful treatments for some forms of cancer, especially blood cancers. By harnessing the same underlying concept — rewiring patients’ own T cells to endow them with therapeutic properties — scientists are working to develop novel CAR-T therapies for a variety of autoimmune diseases. Several research teams are engineering CAR-T cells so they can seek out and destroy harmful immune cells, such as those that produce auto-antibodies — immune proteins that help coordinate the attack on the body’s own tissues. For example, one team is using CAR-T cells to destroy certain immune cells, called B cells, as a potential treatment for lupus, a serious autoimmune disease that mainly affects women. Scientists are also 05 May 2022 3 I NSTI TUTE developing CAR-T therapies that take aim at other rogue members of the immune system. These efforts could yield novel treatments for multiple sclerosis and type 1 diabetes. Regrowing cells in the inner ear to treat hearing loss In the U.S. alone, some 37 million people suffer from a hearing deficit. Currently, there are no drugs that can halt, prevent, or even reverse hearing loss. Scientists are working on a novel regenerative approach that could restore the cells in the inner ear required for normal hearing, offering hope to millions of patients who grapple with hearing loss. Healthy hearing requires specialized cells in the inner ear called hair cells, which have fine, hair-like projections. If the cells are damaged or lost, which often happens with age or after repeated exposure to loud sounds, the body cannot repair them. But researchers have discovered a potential workaround that can stimulate existing cells in the ear to proliferate and give rise to new hair cells. Scientists are now working to convert this molecular strategy, which is being studied in animal models, into a therapeutic that is safe and effective for hearing loss patients. New technologies for delivering gene therapies A formidable challenge in the field of gene therapy is delivery — getting gene-based therapeutics into the body and into the right target cells. Researchers are exploring the potential of new delivery methods that could expand the reach of gene therapy, including microneedles. When applied to the skin, a microneedle patch can penetrate the outermost layer with minimal pain and discomfort. This novel delivery method can readily access the legion of immune cells that reside in the skin — important targets for vaccines as well as for the treatment of various diseases, including cancer and autoimmune conditions. Another emerging technology involves an implantable device made of biodegradable materials. When placed inside the body, this device can provide localized, sustained release of therapeutics with few side effects. The approach is now being tested for the first time in cancer patients using standard chemotherapy drugs administered directly at tumor sites. In the future, this method could be customized for the delivery of gene therapy payloads, an advance that could revolutionize cancer treatment, particularly for difficult-to-treat forms like pancreatic cancer. Engineering cancer-killing cells that target solid tumors CAR-T cells are a revolutionary form of cell therapy that has yielded some remarkable cures of difficult-to-treat blood cancers. But the outcomes in other cancers have been lackluster. Now, scientists are enhancing this technology to enable new ways of treating solid tumors. One approach involves making CAR-T cells more like computers, relying on simple logic to decide which cells are cancer — and should be destroyed — and which cells are healthy and should be spared. By building several logic gates and combining them together, researchers are hoping to pave the way toward targeting new tumor types. Scientists are also devising other groundbreaking forms of cancer-killing cell therapy, including one that uses cancer cells themselves. This approach exploits a remarkable feature: once disseminated within the body, cancer cells can migrate back to the original tumor. Researchers are now harnessing this rehoming capability and, with the help of gene editing, turning tumor cells into potent cancer killers. An early version of this technology uses patients’ own cells. Now, the scientists are developing an off-the-shelf version that can be universally applied to patients. Reawakening the X-chromosome: a therapeutic strategy for devastating neurodevelopmental diseases The X chromosome is one of two sex-determining chromosomes in humans, and it carries hundreds of disease-causing genes. These diseases often affect males and females differently. In females, one X chromosome is naturally, and randomly, chosen and rendered inactive. Although X-inactivation was once thought to be permanent, scientists are uncovering ways to reverse it. Scientists are now exploiting this unusual biology to reawaken the dormant X chromosome — a strategy that could yield muchneeded treatments for a group of rare, yet devastating neurodevelopmental disorders, which predominantly affect females. This new approach could hold promise for females with Rett syndrome, a severe X-linked disorder. A similar strategy could also hold promise for other serious X-linked disorders, including fragile X syndrome and CDKL5 syndrome.

SOURCE

https://business.bofa.com/content/dam/flagship/bank-of-america-institute/transformation/world-innovation-forum-takeaways-may-2022.pdf

Other related articles published in this Open Access Online Scientific Journal include the following:

UPDATED on 5/7/2022

Tweets at #WMIF2022 by @pharma_BI & @AVIVA1950 and All Retweets of these Tweets – 2022 World Medical Innovation Forum, GENE & CELL THERAPY • MAY 2–4, 2022 • BOSTON

Real Time coverage: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2022/05/07/tweets-at-wmif2022-by-pharma_bi-aviva1950-and-all-retweets-of-these-tweets-2022-world-medical-innovation-forum-gene-cell-therapy-may-2-4-2022/

 

2022 World Medical Innovation Forum, GENE & CELL THERAPY • MAY 2–4, 2022 • BOSTON • IN-PERSON

https://pharmaceuticalintelligence.com/2022/05/01/2022-world-medical-innovation-forum-gene-cell-therapy-may-2-4-2022-boston-in-person/

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2022 World Medical Innovation Forum, GENE & CELL THERAPY • MAY 2–4, 2022 • BOSTON • IN-PERSON

Reporter: Aviva Lev-Ari, PhD, RN

World Medical Innovation Forum as we bring together global leaders to assess the latest opportunities and challenges, from the investment landscape to key technology developments to manufacturing and regulatory barriers. Gain first-hand insights on medicine’s ultimate game changer.

https://worldmedicalinnovation.org/

View all videos on youtube.com

https://www.youtube.com/channel/UCauKpbsS_hUqQaPp8EVGYOg

View all tweets on Twitter.com

#WMIF2022

@MGBInnovation

@MassGenBrigham

@pharma_BI

@AVIVA1950

Mass General Brigham Innovation Discovery Grants Program

https://innovation.massgeneralbrigham.org/about/special-programs/partners-innovation-development-grants-program

World Medical Innovation Forum Videos

https://www.youtube.com/channel/UCauKpbsS_hUqQaPp8EVGYOg

World Medical Innovation Forum will be held June 12 – 14 in Boston, MA. We hope you’ll join us for #WMIF2023!

From: “Rieck, Lucy (BOS-WSW)” <LRieck@webershandwick.com>
Date: Tuesday, April 12, 2022 at 10:25 AM
To: Aviva Lev-Ari <avivalev-ari@alum.berkeley.edu>
Subject: You’re Invited: Mass General Brigham’s World Medical Innovation Forum

Hi Aviva,

I’m reaching out to extend free registration for you or a colleague to the 8th annual World Medical Innovation Forum (WMIF), taking place May 2-4 at the Westin Copley Place in Boston. This year’s event, co-sponsored with Bank of America, will explore gene and cell therapies (GCT), including the latest opportunities and challenges – from the investment landscape to key technology developments to manufacturing and regulatory barriers.

The event will feature 200 speakers – including CEOs of leading companies in the GCT and biotech fields, investors, entrepreneurs, Harvard clinicians and scientists, government officials and other key influencers – who discover, invest in, and cultivate GCT breakthroughs. Notable speakers include:

  • Peter Marks: Director, Center for Biologics Evaluation and Research at the FDA
  • Brian Moynihan: CEO, Bank of America
  • Anne Klibansky: President & CEO, Mass General Brigham
  • Senior executives from biopharma and academic institutions of all sizes (including Novartis, BMS, Takeda, Verve, UPenn)

 

You can view the full list of speakers here and the program agenda here.

WMIF is hosted by the Mass General Brigham health system, which comprises 14 hospitals, including two world-renowned medical centers: Mass General and Brigham & Women’s. Since 2015, the Forum has brought together global leaders to assess medical breakthroughs, the investment landscape and technology developments that have the potential to transform the industry.

In addition to a packed agenda, the 2022 “Disruptive Dozen” – 12 breakthrough technologies most likely to have significant impact on gene and cell therapy in the next 18 months – will also be announced.

Please let me know if you would be interested in attending.

Best,

Lucy 

Lucy Rieck

Senior Associate, Healthcare

C: +1 203-331-7894

33 Arch Street

Boston, MA, 02109

webershandwick.com

Ad Age Agency A-List (2020)

Ad Age Best Place to Work (2019)

PRovoke Global Agency of the Decade (2020)

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www.linkedin.com/in/lucyrieck

AGENDA

7:00 AM – 5:00 PMAmerica Foyer
7:00 AM – 8:00 AMAmerica Foyer
8:00 AM – 9:30 AMAmerica Ballroom

FIRST LOOK

First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies

New Gene and Cell Therapy technologies

Moderators:
Meredith Fisher, PhD
  • Partner, Mass General Brigham Ventures
Roger Kitterman
  • VP, Mass General Brigham Ventures
Presenters:
Bakhos Tannous, PhD
  • Director, Experimental Therapeutics Unit, Director, Viral Vector Core, MGH
  • Professor of Neurology, HMS
Vijaya Ramesh, PhD
  • Co-Director of Neuroscience, Associate Geneticist in Neurology, MGH
  • Professor of Neurology, HMS
Anna Krichevsky, PhD
  • Associate Professor of Neurology, BWH, HMS
Nerea Zabaleta, PhD
  • Principal Investigator, Grousbeck Gene Therapy Center, Mass Eye and Ear
  • Instructor in Ophthalmology, HMS
Francisco Quintana, PhD
  • Professor, Neurology, Ann Romney Center for Neurologic Diseases, BWH
  • Kuchroo Weiner Distinguished Professor of Neuroimmunology, BWH
Stephen Haggarty, PhD
  • Director, Chemical Neurobiology Laboratory, Center for Genomic Medicine, MGH
  • Associate Professor of Neurology, HMS
Michael Young, PhD
  • Director, Minda de Gunzburg Center for Retinal Regeneration, Associate Scientist, Schepens Eye Research Institute, Mass Eye and Ear
  • Associate Professor of Ophthalmology, Co-Director, Ocular Regenerative Medicine Institute, HMS
Max Jan, MD, PhD
  • Principal Investigator, Center for Cancer Research, MGH
  • Assistant Professor of Pathology, HMS
9:30 AM – 9:45 AM
9:45 AM – 11:15 AMAmerica Ballroom

FIRST LOOK

First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies

New Gene and Cell Therapy technologies

Moderators:
Meredith Fisher, PhD
  • Partner, Mass General Brigham Ventures
Roger Kitterman
  • VP, Mass General Brigham Ventures
Presenters:
Choi-Fong Cho, PhD
  • Assistant Professor of Neurosurgery, BWH, HMS
Yulia Grishchuk, PhD
  • Assistant Investigator, Center for Genomic Medicine, MGH
  • Assistant Professor of Neurology, HMS
Lynn Bry, MD, PhD
  • Director, Massachusetts Host-Microbiome Center, BWH
  • Associate Professor of Pathology, HMS
David Corey, PhD
  • Bertarelli Professor of Translational Medical Science, Neurobiology, HMS
Anil Chandraker, MD
  • Medical Director of Kidney and Pancreas Transplantation, BWH
  • Associate Professor of Medicine, HMS
Ole Isacson, MD, PhD
  • Director, Neuroregeneration Research Institute, McLean
  • Professor of Neurology & Neuroscience, HMS
Marco Mineo, PhD
  • Instructor in Neurosurgery, BWH, HMS
Susan Cotman, PhD
  • Assistant in Neuroscience, Center for Genomic Medicine, MGH
  • Assistant Professor of Neurology, HMS
11:15 AM – 11:45 AM
11:45 AM – 12:45 PM3rd Floor and 7th Floor

DR. IS IN

Dr. Is In Sessions

Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.

1:00 PM – 1:30 PMAmerica Ballroom

Opening Remarks

Introducer:
Scott Sperling
  • Co-Chief Executive Officer, Thomas H. Lee Partners
  • Chairman of the Board of Directors, Mass General Brigham
Panelists:
Anne Klibanski, MD
  • President & CEO, Mass General Brigham
  • Laurie Carrol Guthart Professor of Medicine, HMS
Brian Moynihan
  • Chair & CEO, Bank of America
1:30 PM – 2:00 PMAmerica Ballroom

Co-Chair Kick Off

Moderator:
Susan Hockfield, PhD
  • President Emerita, MIT
Panelists:
Miceal Chamberlain
  • President of Massachusetts, Northeast Region Executive, Bank of America
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
2:00 PM – 2:40 PMAmerica Ballroom

GCT’s Historic Potential | Priorities and Trade Offs

This panel features industry leaders who will discuss what the future may hold for gene and cell therapy. Which applications are likely to have the greatest impact? What are the key hurdles to be overcome? What specific platforms and technologies may enable optimal solutions? In what disease areas? Learn more about these and other questions as the panelists discuss the future potential of GCT.

Moderator:
Jean-François Formela, MD
  • Partner, Atlas Venture
Panelists:
Pablo Cagnoni, MD
  • CEO, Rubius Therapeutics
Kristen Hege, MD
  • Senior Vice President, Early Clinical Development, Hematology/Oncology & Cell Therapy, Bristol Myers Squibb
Andrew Plump, MD, PhD
  • President, R&D, Takeda
Catherine Stehman-Breen, MD
  • CEO, Chroma Medicine
2:40 PM – 3:20 PMAmerica Ballroom

Manufacturing | Process Control

Manufacturing quality and cost are critical for enabling rapid growth in GCT. Panelists will explore a variety of critical questions in this space. For example, are there historic parallels that can be drawn between GCT manufacturing and other groundbreaking technologies? How do key manufacturing concerns in GCT differ from those for more conventional pharmaceutical? What are the long-term opportunities for non-viral vectors? Will manufacturing capacity be a limiting factor in GCT growth over the next 5 to 10 years?

Moderator:
John Bishai, PhD
  • Managing Director, Global Investment Banking, BofA Securities
Panelists:
Christopher Murphy
  • Vice President Viral Vector Services, Thermo Fisher
Michael Paglia
  • COO, ElevateBio BaseCamp, ElevateBio
Rahul Singhvi, ScD
  • CEO, National Resilience, Inc.
Ran Zheng
  • CEO, Landmark Bio
3:20 PM – 3:40 PM
3:40 PM – 4:05 PMAmerica Ballroom

FIRESIDE

Regulatory Perspectives on Gene and Cell Therapy: Past Lessons, Current Challenges, Future Directions

At the end of 2021, roughly 410 novel drugs had been approved in the past decade. On average, there were 40 approvals per year with over 150 of them being between 2018 and 2020. What has changed in the approval process and what is the vision of the future state? What will happen over the next 1–3 years? What does the new iteration of the Prescription Drug User Fees Act (PDUFA) need to do in this area and which fields show the greatest potential for innovation in CGT?

Moderator:
Luk Vandenberghe, PhD
  • Grousbeck Associate Professor in Gene Therapy, Mass General Brigham (on leave)
Panelist:
Peter Marks, MD, PhD
  • Director, Center for Biologics Evaluation and Research, FDA
4:10 PM – 4:50 PMAmerica Ballroom

Clinical GCT Trial Design | Regulatory | Strategy, Innovation and Future Direction | Risk vs Hype

This panel will delve into clinical trials for GCT. How do these trials differ from those for conventional therapeutics? What are the key lessons learned from completed GCT trials? How is the regulatory landscape shifting and what will that mean for the future of GCT?

Moderator:
Angela Shen, MD
  • Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
Panelists:
Laura Aguilar, MD, PhD
  • Co-Founder, Candel Therapeutics
Matthew Frigault, MD
  • Clinical Director, Cellular Immunotherapy Program, MGH
  • Assistant Professor of Medicine, HMS
Arati Rao, MD
  • Senior Vice President, Clinical Development, PACT Pharma
John Rossi
  • VP Head of Translational Medicine, Syncopation Life Sciences
4:50 PM – 5:15 PMAmerica Ballroom

FIRESIDE

mRNA Opportunities: Lessons Learned, Priorities, and the Future of GCT

Dr. Bourla will share what Pfizer has learned from its leadership on mRNA and the development of the Covid vaccine that can be extrapolated to other R&D.

Moderator:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Albert Bourla, PhD
  • CEO, Pfizer Inc.
5:15 PM – 6:15 PMAmerica Foyer

#WMIF2022

@MGBInnovation

@MassGenBrigham

@pharma_BI

@AVIVA1950

7:00 AM – 5:00 PMAmerica Foyer
7:00 AM – 8:00 AMAmerica Foyer

Breakfast

Sponsored by Bayer

7:45 AM – 8:00 AMAmerica Ballroom

Opening Remarks

Introducer:
Chris Coburn
  • Chief Innovation Officer, Mass General Brigham
8:00 AM – 8:25 AMAmerica Ballroom

FIRESIDE

1:1 Fireside Chat: Robert Califf, MD, Commissioner Food and Drugs, FDA

Moderators:
Tazeen Ahmad
  • Managing Director, Global Research, BofA Securities
J. Keith Joung, MD, PhD
  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS
Panelist:
Robert Califf, MD
  • Commissioner of Food and Drugs, US Food and Drug Administration
8:25 AM – 9:05 AMAmerica Ballroom

Living with COVID | Lessons Learned and Looking Ahead

As we enter the third year of the coronavirus pandemic, the world is shifting to a new strategy: living with and managing COVID as a part of our everyday lives. What will the coming year look like? How will mitigation measures differ in this new phase? What about treatment strategies? Should we be bracing for another surge?

Introducer:
Jonathan Kraft
  • President, The Kraft Group
  • Chairman of the Board of Trustees, MGH
Moderator:
David Brown, MD
  • President, Massachusetts General Hospital
  • Executive Vice President, Mass General Brigham
Panelists:
Paul Biddinger, MD
  • Chief Preparedness and Continuity Officer, Mass General Brigham
  • Associate Professor of Emergency Medicine, HMS
Helen Branswell
  • Senior Writer, STAT
Daniel Kuritzkes, MD
  • Chief, Division of Infectious Diseases, BWH
  • Harriet Ryan Albee Professor of Medicine, HMS
Erica Shenoy, MD, PhD
  • Associate Chief, Infection Control Unit, MGH
  • Associate Professor of Medicine, HMS
9:05 AM – 9:45 AMAmerica Ballroom

The Global Biotech Epicenter | New England Now and in 2030

This panel will feature a discussion of global biotech clusters with a deep dive into the New England/Boston area. How does the capital availability, scale, and density of New England drive local growth in GCT? Also, the influx of large biopharmaceutical companies into the region has fueled global outcomes. What is the future impact of these investments and when will they peak? How will the biopharmaceutical landscape in New England appear in 2030?

Moderator:
Anne Finucane
  • Chairman of the Board, Bank of America Europe
Panelists:
Seth Ettenberg, PhD
  • President & CEO, BlueRock Therapeutics
Joel Marcus
  • Executive Chairman & Founder, Alexandria Real Estate Equities, Inc.
Terry McGuire
  • Founding Partner, Polaris Partners
Vicki Sato, PhD
  • Chairman of the Board, Vir Biotechnology
  • Chairman, Denali Therapeutics
Phillip Sharp, PhD
  • Institute Professor and Professor of Biology, Koch Institute for Integrative Cancer Research at MIT
  • Co-Founder, Alnylam Pharmaceuticals, Inc.
9:45 AM – 10:05 AM
10:10 AM – 10:50 AMAmerica Ballroom

The Patient Experience

The role of patients and their experiences are critical as the promise of GCT unfolds. This panel will discuss the patient experience and explore the challenges different patient populations face, both in rare diseases and more common conditions. Panelists will also discuss financial considerations, clinical trial access, and the role of advocacy groups in GCT.

Moderator:
Merit Cudkowicz, MD
  • Chair, Dept of Neurology, MGH
  • Julieanne Dorn Professor of Neurology, HMS
Panelist:
James Beck, PhD
  • CSO, Parkinson’s Foundation
Monica Coenraads
  • CEO, Rett Syndrome Research Trust
Annie Ganot
  • VP, Head of Patient Advocacy, Solid Biosciences
Staci Kallish, DO
  • President, Board of Directors, National Tay Sachs and Allied Diseases
  • Medical Geneticist, Associate Professor of Clinical Medicine, Penn Medicine
Rebecca Oberman, PhD
  • Executive Director, Mucolipidosis Type IV (ML4) Foundation
10:50 AM – 11:15 AMAmerica Ballroom

FIRESIDE

Meeting the Moment: The Next Wave of Innovation in Cancer and Cardiology

As many countries begin to turn the corner on COVID-19, they face a resurgence of chronic illnesses, such as cancer and cardiovascular disease, that were not adequately addressed during the pandemic, and for which new treatments are urgently needed. Population aging – and the resulting increase in chronic diseases associated with aging – has compounded the challenge. There’s never been a greater need for biopharmaceutical innovation – or, fortunately, a greater ability to innovate. Amgen is investing in new discovery research capabilities that portend a revolution in drug design and development.

Moderator:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Robert Bradway
  • CEO, Amgen
11:15 AM – 11:20 AMAmerica Ballroom

First Look Award Presentation

Presenters:
Miceal Chamberlain
  • President of Massachusetts, Northeast Region Executive, Bank of America
Nino Chiocca, MD, PhD
  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS
11:20 AM – 11:30 AMAmerica Ballroom
11:30 AM – 11:45 AM
11:45 AM – 12:45 PM3rd Floor and 7th Floor

DR. IS IN

Dr. Is In Sessions

Lunch Sponsored by Astellas

Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.

  • Personalizing Cancer Care through RNA Therapies

    11:45 AM – 12:45 PM

    In this session, Dr. Peruzzi will discuss how RNA for cancer therapy is a versatile of a tool for a protean problem.

    Moderator:
    Jason Gerberry
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Pierpaolo Peruzzi, MD, PhD
    • Neurosurgeon and Principal Investigator, BWH
    • Assistant Professor of Neurosurgery, HMS
  • Designing for Success: Clinical Trial Approaches for Rare and Ultra-Rare Diseases

    11:45 AM – 12:45 PM

    In this session, Dr. Vavvas will discuss examples of clinical trials in rare diseases and share insights into how clinical trials should be approached for rare and ultra-rare diseases and how study design is not a one-size fits all.

    Moderator:
    Tazeen Ahmad
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Demetrios Vavvas, MD, PhD
    • Associate Director of the Retina Service, Mass Eye and Ear
    • Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS
  • A New Hope: Cell Therapy and Transplantation for Parkinson’s Disease

    11:45 AM – 12:45 PM

    In this session, hear experts weigh in on the possibilities of cell therapy development and transplantation for the treatment of Parkinson’s Disease. What does the futures hold and how do we get there?

    Moderator:
    Greg Harrison
    • Vice President, Global Research, BofA Securities
    Panelist:
    Bob Carter, MD, PhD
    • Chairman, Department of Neurosurgery, MGH
    • William and Elizabeth Sweet Professor of Neurosurgery, HMS
    Todd Herrington, MD, PhD
    • Director, Deep Brain Stimulation Program, MGH
    • Assistant Professor of Neurology, HMS
    Kwang-Soo Kim, PhD
    • Director, Molecular Neurobiology Laboratory, McLean
    • Professor of Neuroscience and Psychiatry, HMS
    Jeffrey Schweitzer, MD, PhD
    • Neurosurgeon, MGH
    • Assistant Professor of Neurosurgery, HMS
  • The Inner Workings of Gene Therapy Manufacturing

    11:45 AM – 12:45 PM

    In this session, Dr. Nikiforow will provide insights into the world of gene therapy manufacturing and the complexities of scaling, costs and insurance reimbursement.

    Moderator:
    Michael Ryskin
    • Director, Global Research, BofA Securities
    Panelist:
    Sarah Nikiforow, MD, PhD
    • Medical Director, Cell Manipulation Core Facility, Technical Director, Immune Effector Cell Therapy Program, DFCI
    • Assistant Professor, HMS
  • The Road Ahead: Regulatory Challenges for Gene and Cell Therapy

    11:45 AM – 12:45 PM

    In this session, Dr. Marks will discuss the ins and outs of regulatory challenges for biological products and therapies in gene and cell therapy and the responsibility to assure safety and effectiveness.

    Moderator:
    Geoff Meacham, PhD
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Peter Marks, MD, PhD
    • Director, Center for Biologics Evaluation and Research, FDA
  • The Mysterious Dark Genome

    11:45 AM – 12:45 PM

    Dark genome, accounting for ~98.5% of the human genome and containing the non-coding part, offers unprecedented opportunity to look for novel elements that could play a role in human health. This non-coding region consists of repeat elements, enhancers, regulatory sequences and non-coding RNAs. This session will explore this exciting new frontier in biology and how to translate this so called “junk” and previously ignored genome into potential novel therapeutics.

    Moderators:
    Angela Shen, MD
    • Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
    Richard Young, PhD
    • Professor, Whitehead Institute, MIT
    Panelists:
    Rosana Kapeller, MD, PhD
    • Co-Founder, President & CEO, ROME Therapeutics
    Josh Mandel-Brehm
    • President & CEO, CAMP4 Therapeutics
    Amir Nashat, PhD
    • Managing Partner, Polaris Ventures
    Issi Rozen
    • Venture Partner, GV
1:00 PM – 1:40 PMAmerica Ballroom

Capital Formation | Shaping Innovation

Panelists will discuss the life sciences capital markets environment with particular emphasis on private and public fundraising for GCT companies. What trends do panelists observe that will impact the availability and cost of capital for GCT? Are there novel fundraising structures that will serve GCT in the future?

Moderator:
Greg Butz
  • Managing Director, Head of Life Sciences Investment Banking, BofA Securities
Sumit Mukherjee
  • Managing Director & Head of Healthcare in Equity Capital Markets, BofA Securities
Panelists:
Shelley Chu, MD, PhD
  • Partner, Lightspeed
Stephen Knight, MD
  • President & Managing Partner, F-Prime Capital
Adam Koppel, MD, PhD
  • Managing Director, Bain Capital Life Sciences
Daniel Krizek
  • Portfolio Manager, Citadel
1:40 PM – 2:05 PMAmerica Ballroom

FIRESIDE

Ending Cancer as We Know It: The Game Changing Potential of GCT

50 years after the nation’s War on Cancer was launched, do new treatment innovations have us at a turning point to end cancer “as we know it”.

Moderator:
Erin Harris
  • Chief Editor, Cell & Gene
Panelists:
David Scadden, MD
  • Director, Center for Regenerative Medicine, MGH
  • Gerald and Darlene Jordan Professor of Medicine, HMS
Norman Sharpless, MD
  • Former Director, National Cancer Institute
2:05 PM – 2:30 PMAmerica Ballroom

FIRESIDE

Vision and Execution: Curing Disease with Cell Therapies

As one of the foremost researchers of CAR-T cancer treatments, Dr. June will share what he believes is the next wave of cell-and-gene based oncology research and how his work set the stage for breakthrough developments in cancer.

Moderators:
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelist:
Carl June, MD
  • Richard W. Vague Professor in Immunotherapy, Director, Center for Cellular Immunotherapies, Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
2:30 PM – 3:10 PMAmerica Ballroom

GCT Development Centers | Academia’s Unique Contribution

This panel will examine the role of academia in driving the promise of GCT. How does academic innovation contribute to the success of GCT? What are the risks and opportunities? Which models have proven most successful and what is the impact on clinical translation? How can these partnerships be accelerated?

Moderator:
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelists:
Carl June, MD
  • Richard W. Vague Professor in Immunotherapy, Director, Center for Cellular Immunotherapies, Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
Maria Millan, MD
  • President & CEO, California Institute for Regenerative Medicine
Richard Mulligan, PhD
  • Mallinckrodt Professor of Genetics, Emeritus, HMS
  • Executive Vice Chairman, Sana Biotechnology, Inc
Norman Sharpless, MD
  • Former Director, National Cancer Institute
3:10 PM – 3:30 PM
3:30 PM – 3:55 PMAmerica Ballroom

FIRESIDE

1:1 Fireside Chat: Marc Casper

Moderator:
Derik de Bruin, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Marc Casper
  • CEO, ThermoFisher
3:55 PM – 4:35 PMAmerica Ballroom

Gene and Cell Therapy | The World Speaks

This panel will bring together gene and cell therapy leaders from across the world to discuss the latest opportunities and challenges in the field, from the investment landscape to key technology developments to manufacturing and regulatory barriers. These global experts will offer first-hand insights on the systemic complexity of this advancing field and its therapeutic promise.

Moderator:
Christine Fox
  • President, Novartis Gene Therapies
Panelists:
Christopher Baum, MD
  • Chairman of the Board of Directors, Berlin Institute of Health
Nicholas Galakatos, PhD
  • Global Head of Life Sciences, Blackstone
Luigi Naldini, MD, PhD
  • Director, San Raffaele Telethon Institute for Gene Therapy
Kendra Rose, PhD
  • VP, Head of New Platforms, Ophthalmology and Hemophilia, Bayer
4:35 PM – 5:15 PMAmerica Ballroom

Control or Mitigation of the Effects of Chronic Neuroinflammation

Chronic inflammation in the brain is now recognized as a contributor to many neurodegenerative diseases, ranging from Parkinson’s disease to multiple sclerosis to Alzheimer’s disease. Are solutions to these historically intractable neurological diseases imminent or several years away? Are market-making platforms identifiable for neurological diseases? Are there novel genetic targets that can be explored? What are the prospects for cell therapies?

Moderator:
Ole Isacson, MD, PhD
  • Director, Neuroregeneration Research Institute, McLean
  • Professor of Neurology & Neuroscience, HMS
Panelists:
Colin Hill
  • CEO, GNS Healthcare
Spyros Papapetropoulos, MD, PhD
  • CMO, Vigil Neuroscience
Richard Ransohoff, MD
  • CMO, Abata Therapeutics
  • Venture Partner, Third Rock Ventures
Beth Stevens, PhD
  • HHMI Investigator, F.M. Kirby Neurobiology Research Program, Boston Children’s Hospital
  • Associate Professor of Neurology, HMS
Rudolph Tanzi, PhD
  • Vice-Chair, Neurology, Director, Genetics and Aging Research Unit, MGH
  • Joseph P. and Rose F. Kennedy Professor of Neurology, HMS
5:15 PM – 6:15 PMAmerica Foyer

Attendee Networking Reception

Sponsored by Novartis

#WMIF2022

@MGBInnovation

@MassGenBrigham

@pharma_BI

@AVIVA1950

7:00 AM – 12:00 PMAmerica Foyer
7:00 AM – 8:00 AMAmerica Foyer
8:05 AM – 8:45 AMAmerica Ballroom

The Cell Therapy Landscape | CAR-T to Stem Cells

Cell therapies, ranging from CAR-T cells to stem-cell-based approaches, are emerging as a transformative therapeutic modality. Panelists will examine this emerging landscape and discuss a range of key topics. What drives differentiation in this space given the high number of competing technologies? How will the uptake of autologous cell therapies and allogeneic versions evolve? When will the regenerative medicine market mature?

Moderator:
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Panelists:
Christina Coughlin, MD, PhD
  • CEO, Cytoimmune
Rachel Haurwitz, PhD
  • President & CEO, Caribou Biosciences
Nick Leschly
  • CEO, 2seventy bio
Dhvanit Shah, PhD
  • President & CEO, Garuda Therapeutics
Rusty Williams, MD, PhD
  • Chairman & CEO, Walking Fish Therapeutics
8:50 AM – 9:30 AMAmerica Ballroom

Disrupting Interventions

This panel will explore how GCT technology could lead to disruptions in other areas of medicine, including surgery and medical devices, over the next several years. Could cell replacement therapy in diabetes advance enough to reduce the need for diabetes pumps or insulin? Will stem-cell-based methods for regenerating cartilage advance rapidly enough to disrupt the number of patients seeking hip and knee replacements? How is GCT driving innovations in surgical techniques?

Introducer:
John Fish
  • Chairman & CEO, Suffolk
  • Chair, Brigham and Women’s Hospital
Moderator:
Robert Higgins, MD
  • President, Brigham and Women’s Hospital
  • Executive Vice President, Mass General Brigham
Panelists:
Irina Antonijevic, MD, PhD
  • CMO and Head of R&D, Triplet Therapeutics, Inc.
Rachel McMinn, PhD
  • Founder & CEO, Neurogene
Harith Rajagopalan, MD, PhD
  • CEO & Co-Founder, Fractyl Health
Bastiano Sanna, PhD
  • EVP, Chief of Cell & Gene Therapies and VCGT Site Head, Vertex Pharmaceuticals
Jeffrey Schweitzer, MD, PhD
  • Neurosurgeon, MGH
  • Assistant Professor of Neurosurgery, HMS
9:30 AM – 9:55 AMAmerica Ballroom

FIRESIDE

1:1 Fireside Chat: Dan Skovronsky

Moderator:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Daniel Skovronsky, MD, PhD
  • Chief Scientific and Medical Officer, Eli Lilly and Company
9:55 AM – 10:20 AMAmerica Ballroom

FIRESIDE

Reimagining GCT Production

What is the new generation of approaches to gene therapy manufacturing and delivery? What are the lessons learned from Covid and how can it be applied to custom disease response and the ability to custom design biologic organisms?

Moderator:
Derik de Bruin, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Jason Kelly, PhD
  • Co-Founder & CEO, Ginkgo Bioworks
10:20 AM – 11:00 AMAmerica Ballroom

Gene and Cell Therapy Safety | Enduring Framework Required

This panel will feature an in-depth discussion of the safety of gene and cell therapies. What are the unique safety concerns in this field, both acute and potential long-term risks? Which of these concerns are supported by clinical data versus the presumption of theoretical risk? What are the key issues for AAV-based gene therapies? Will redosing become feasible? What are the predominant safety concerns for in vivo versus ex vivo GCT modalities, including base editing?

Moderator:
Christine Seidman, MD
  • Director, Cardiovascular Genetics Center, BWH
  • Smith Professor of Medicine & Genetics, HMS
Panelists:
Rick Fair
  • President & CEO, Bellicum
Alexandria Forbes, PhD
  • President & CEO, MeiraGTx
Sekar Kathiresan, MD
  • CEO, Verve Therapeutics
Rick Modi
  • CEO, Affinia Therapeutics
11:00 AM – 11:40 AMAmerica Ballroom

RNA Therapeutics | Lessons Learned

The label “RNA” encompasses a wide array of biologically active agents spanning therapeutic modalities, vaccines, non-coding controls, and other forms. In this panel we will discuss a number of these forms, discuss examples of recent developments and illustrate why RNA developments represent a promising source of novel therapies and therapeutic approaches.

Moderator:
Janet Wu
  • Anchor/Reporter, Bloomberg
Panelists:
Sarah Boyce
  • President & CEO, Avidity Biosciences, Inc.
Jim Burns, PhD
  • CEO, Locanabio
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
Laura Sepp-Lorenzino, PhD
  • Chief Scientific Officer, Executive Vice President, Intellia Therapeutics
11:40 AM – 12:40 PMAmerica Ballroom

Disruptive Dozen: 12 Technologies That Will Reinvent GCT in the Next Five Years

The Disruptive Dozen identifies and ranks the GCT technologies that Mass General Brigham faculty feel will break through over the next one to five years to significantly improve health care.

Moderators:
Nino Chiocca, MD, PhD
  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS
Susan Slaugenhaupt, PhD
  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr. Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelists:
Galit Alter, PhD
  • Principal Investigator, Ragon Institute, MGH
  • Professor of Medicine, HMS
Natalie Artzi, PhD
  • Assistant Professor of Medicine, HMS
Fengfeng Bei, PhD
  • Principal Investigator, Department of Neurosurgery, BWH
  • Assistant Professor of Neurosurgery, HMS
Zheng-Yi Chen, DPhil
  • Associate Scientist, Eaton-Peabody Laboratories, Mass Eye and Ear
  • Associate Professor of Otolaryngology Head and Neck Surgery, HMS
Matthew Frigault, MD
  • Clinical Director, Cellular Immunotherapy Program, MGH
  • Assistant Professor of Medicine, HMS
Michael Gilmore, PhD
  • Chief Scientific Officer, Mass Eye and Ear
  • Sir William Osler Professor of Ophthalmology, HMS
Allan Goldstein, MD
  • Chief of Pediatric Surgery, MGH
  • Surgeon-in-Chief, MassGeneral for Children
Anna Krichevsky, PhD
  • Associate Professor of Neurology, BWH, HMS
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
James Markmann, MD, PhD
  • Chief, Division of Transplant Surgery, MGH
  • Claude E. Welch Professor of Surgery, HMS
Khalid Shah, PhD
  • Vice Chairman of Research, Department of Neurosurgery, BWH
  • Professor, HMS
Demetrios Vavvas, MD, PhD
  • Associate Director of the Retina Service, Mass Eye and Ear
  • Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS

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Bipolar Disorder now understood by Markers Identified of the Gene Expression for this Diagnosis

Reporter: Aviva Lev-Ari, PhD, RN

Published: 

Amygdala and anterior cingulate transcriptomes from individuals with bipolar disorder reveal downregulated neuroimmune and synaptic pathways

Abstract

Recent genetic studies have identified variants associated with bipolar disorder (BD), but it remains unclear how brain gene expression is altered in BD and how genetic risk for BD may contribute to these alterations. Here, we obtained transcriptomes from subgenual anterior cingulate cortex and amygdala samples from post-mortem brains of individuals with BD and neurotypical controls, including 511 total samples from 295 unique donors. We examined differential gene expression between cases and controls and the transcriptional effects of BD-associated genetic variants. We found two coexpressed modules that were associated with transcriptional changes in BD: one enriched for immune and inflammatory genes and the other with genes related to the postsynaptic membrane. Over 50% of BD genome-wide significant loci contained significant expression quantitative trait loci (QTL) (eQTL), and these data converged on several individual genes, including SCN2A and GRIN2A. Thus, these data implicate specific genes and pathways that may contribute to the pathology of BP.

SOURCE

https://www.nature.com/articles/s41593-022-01024-6

Gene Expression Markers for Bipolar Disorder Pinpointed

The work was led by researchers at Johns Hopkins’ Lieber Institute for Brain Development. The findings, published this week in Nature Neuroscience, represent the first time that researchers have been able to apply large-scale genetic research to brain samples from hundreds of patients with bipolar disorder (BD). They used 511 total samples from 295 unique donors.

“This is the first deep dive into the molecular biology of the brain in people who died with bipolar disorder—studying actual genes, not urine, blood or skin samples,” said Thomas Hyde of the Lieber Institute and a lead author of the paper. “If we can figure out the mechanisms behind BD, if we can figure out what’s wrong in the brain, then we can begin to develop new targeted treatments of what has long been a mysterious condition.”

Bipolar disorder is characterized by extreme mood swings, with episodes of mania alternating with episodes of depression. It usually emerges in people in their 20s and 30s and remains with them for life. This condition affects approximately 2.8% of the adult American population, or about 7 million people. Patients face higher rates of suicide, poorer quality of life, and lower productivity than the general population. Some estimates put the annual cost of the condition in the U.S. alone at $219.1 billion.

While drugs can be useful in treating BD, many patients find they have bothersome side effects, and for some patients, current medications don’t work at all.

In this study, researchers measured levels of messenger RNA in the brain samples. They observed almost eight times more differentially expressed gene features in the sACC versus the amygdala, suggesting that the sACC may play an especially prominent role—both in mood regulation in general and BD specifically.

In patients who died with BD, the researchers found abnormalities in two families of genes: one containing genes related to the synapse and the second related to immune and inflammatory function.

“There finally is a study using modern technology and our current understanding of genetics to uncover how the brain is doing,” Hyde said. “We know that BD tends to run in families, and there is strong evidence that there are inherited genetic abnormalities that put an individual at risk for bipolar disorder. Unlike diseases such as sickle-cell anemia, bipolar disorder does not result from a single genetic abnormality. Rather, most patients have inherited a group of variants spread across a number of genes.”

“Bipolar disorder, also known as manic-depressive disorder, is a highly damaging and paradoxical condition,” said Daniel R. Weinberger, chief executive and director of the Lieber Institute and a co-author of the study. “It can make people very productive so they can lead countries and companies, but it can also hurl them into the meat grinder of dysfunction and depression. Patients with BD may live on two hours of sleep a night, saving the world with their abundance of energy, and then become so self-destructive that they spend their family’s fortune in a week and lose all friends as they spiral downward. Bipolar disorder also has some shared genetic links to other psychiatric disorders, such as schizophrenia, and is implicated in overuse of drugs and alcohol.”

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New studies link cell cycle proteins to immunosurveillance of premalignant cells

Curator: Stephen J. Williams, Ph.D.

The following is from a Perspectives article in the journal Science by Virinder Reen and Jesus Gil called “Clearing Stressed Cells: Cell cycle arrest produces a p21-dependent secretome that initaites immunosurveillance of premalignant cells”. This is a synopsis of the Sturmlechener et al. research article in the same issue (2).

Complex organisms repair stress-induced damage to limit the replication of faulty cells that could drive cancer. When repair is not possible, tissue homeostasis is maintained by the activation of stress response programs such as apoptosis, which eliminates the cells, or senescence, which arrests them (1). Cellular senescence causes the arrest of damaged cells through the induction of cyclin-dependent kinase inhibitors (CDKIs) such as p16 and p21 (2). Senescent cells also produce a bioactive secretome (the senescence-associated secretory phenotype, SASP) that places cells under immunosurveillance, which is key to avoiding the detrimental inflammatory effects caused by lingering senescent cells on surrounding tissues. On page 577 of this issue, Sturmlechner et al. (3) report that induction of p21 not only contributes to the arrest of senescent cells, but is also an early signal that primes stressed cells for immunosurveillance.Senescence is a complex program that is tightly regulated at the epigenetic and transcriptional levels. For example, exit from the cell cycle is controlled by the induction of p16 and p21, which inhibit phosphorylation of the retinoblastoma protein (RB), a transcriptional regulator and tumor suppressor. Hypophosphorylated RB represses transcription of E2F target genes, which are necessary for cell cycle progression. Conversely, production of the SASP is regulated by a complex program that involves super-enhancer (SE) remodeling and activation of transcriptional regulators such as nuclear factor κB (NF-κB) or CCAAT enhancer binding protein–β (C/EBPβ) (4).

Senescence is a complex program that is tightly regulated at the epigenetic and transcriptional levels. For example, exit from the cell cycle is controlled by the induction of p16 and p21, which inhibit phosphorylation of the retinoblastoma protein (RB), a transcriptional regulator and tumor suppressor. Hypophosphorylated RB represses transcription of E2F target genes, which are necessary for cell cycle progression. Conversely, production of the SASP is regulated by a complex program that involves super-enhancer (SE) remodeling and activation of transcriptional regulators such as nuclear factor κB (NF-κB) or CCAAT enhancer binding protein–β (C/EBPβ) (4).

Sturmlechner et al. found that activation of p21 following stress rapidly halted cell cycle progression and triggered an internal biological timer (of ∼4 days in hepatocytes), allowing time to repair and resolve damage (see the figure). In parallel, C-X-C motif chemokine 14 (CXCL14), a component of the PASP, attracted macrophages to surround and closely surveil these damaged cells. Stressed cells that recovered and normalized p21 expression suspended PASP production and circumvented immunosurveillance. However, if the p21-induced stress was unmanageable, the repair timer expired, and the immune cells transitioned from surveillance to clearance mode. Adjacent macrophages mounted a cytotoxic T lymphocyte response that destroyed damaged cells. Notably, the overexpression of p21 alone was sufficient to orchestrate immune killing of stressed cells, without the need of a senescence phenotype. Overexpression of other CDKIs, such as p16 and p27, did not trigger immunosurveillance, likely because they do not induce CXCL14 expression.In the context of cancer, senescent cell clearance was first observed following reactivation of the tumor suppressor p53 in liver cancer cells. Restoring p53 signaling induced senescence and triggered the elimination of senescent cells by the innate immune system, prompting tumor regression (5). Subsequent work has revealed that the SASP alerts the immune system to target preneoplastic senescent cells. Hepatocytes expressing the oncogenic mutant NRASG12V (Gly12→Val) become senescent and secrete chemokines and cytokines that trigger CD4+ T cell–mediated clearance (6). Despite the relevance for tumor suppression, relatively little is known about how immunosurveillance of oncogene-induced senescent cells is initiated and controlled.

Source of image: Reen, V. and Gil, J. Clearing Stressed Cells. Science Perspectives 2021;Vol 374(6567) p 534-535.

References

2. Sturmlechner I, Zhang C, Sine CC, van Deursen EJ, Jeganathan KB, Hamada N, Grasic J, Friedman D, Stutchman JT, Can I, Hamada M, Lim DY, Lee JH, Ordog T, Laberge RM, Shapiro V, Baker DJ, Li H, van Deursen JM. p21 produces a bioactive secretome that places stressed cells under immunosurveillance. Science. 2021 Oct 29;374(6567):eabb3420. doi: 10.1126/science.abb3420. Epub 2021 Oct 29. PMID: 34709885.

More Articles on Cancer, Senescence and the Immune System in this Open Access Online Scientific Journal Include

Bispecific and Trispecific Engagers: NK-T Cells and Cancer Therapy

Natural Killer Cell Response: Treatment of Cancer

Issues Need to be Resolved With ImmunoModulatory Therapies: NK cells, mAbs, and adoptive T cells

New insights in cancer, cancer immunogenesis and circulating cancer cells

Insight on Cell Senescence

Immune System Stimulants: Articles of Note @pharmaceuticalintelligence.com

Read Full Post »

The Vibrant Philly Biotech Scene: Proteovant Therapeutics Using Artificial Intelligence and Machine Learning to Develop PROTACs

Reporter: Stephen J. Williams, Ph.D.

It has been a while since I have added to this series but there have been a plethora of exciting biotech startups in the Philadelphia area, and many new startups combining technology, biotech, and machine learning. One such exciting biotech is Proteovant Therapeutics, which is combining the new PROTAC (Proteolysis-Targeting Chimera) technology with their in house ability to utilize machine learning and artificial intelligence to design these types of compounds to multiple intracellular targets.

PROTACs (which actually is under a trademark name of Arvinus Operations, but is also refered to as Protein Degraders. These PROTACs take advantage of the cell protein homeostatic mechanism of ubiquitin-mediated protein degradation, which is a very specific targeted process which regulates protein levels of various transcription factors, protooncogenes, and receptors. In essence this regulated proteolyic process is needed for normal cellular function, and alterations in this process may lead to oncogenesis, or a proteotoxic crisis leading to mitophagy, autophagy and cellular death. The key to this technology is using chemical linkers to associate an E3 ligase with a protein target of interest. E3 ligases are the rate limiting step in marking the proteins bound for degradation by the proteosome with ubiquitin chains.

Model of PROTAC Ternarary Complex

A review of this process as well as PROTACs can be found elsewhere in articles (and future articles) on this Open Access Journal.

Protevant have made two important collaborations:

  1. Oncopia Therapeutics: came out of University of Michigan Innovation Hub and lab of Shaomeng Wang, who developed a library of BET and MDM2 based protein degraders. In 2020 was aquired by Riovant Sciences.
  2. Riovant Sciences: uses computer aided design of protein degraders

Proteovant Company Description:

Proteovant is a newly launched development-stage biotech company focusing on discovery and development of disease-modifying therapies by harnessing natural protein homeostasis processes. We have recently acquired numerous assets at discovery and development stages from Oncopia, a protein degradation company. Our lead program is on track to enter IND in 2021. Proteovant is building a strong drug discovery engine by combining deep drugging expertise with innovative platforms including Roivant’s AI capabilities to accelerate discovery and development of protein degraders to address unmet needs across all therapeutic areas. The company has recently secured $200M funding from SK Holdings in addition to investment from Roivant Sciences. Our current therapeutic focus includes but is not limited to oncology, immunology and neurology. We remain agnostic to therapeutic area and will expand therapeutic focus based on opportunity. Proteovant is expanding its discovery and development teams and has multiple positions in biology, chemistry, biochemistry, DMPK, bioinformatics and CMC at many levels. Our R&D organization is located close to major pharmaceutical companies in Eastern Pennsylvania with a second site close to biotech companies in Boston area.

Protein degradation

Source: Protevant

The ubiquitin proteasome system (UPS) is responsible for maintaining protein homeostasis. Targeted protein degradation by the UPS is a cellular process that involves marking proteins and guiding them to the proteasome for destruction. We leverage this physiological cellular machinery to target and destroy disease-causing proteins.

Unlike traditional small molecule inhibitors, our approach is not limited by the classic “active site” requirements. For example, we can target transcription factors and scaffold proteins that lack a catalytic pocket. These classes of proteins, historically, have been very difficult to drug. Further, we selectively degrade target proteins, rather than isozymes or paralogous proteins with high homology. Because of the catalytic nature of the interactions,  it is possible to achieve efficacy at lower doses with prolonged duration while decreasing dose-limiting toxicities.

Biological targets once deemed “undruggable” are now within reach.

About Riovant Sciences: from PRNewsWire https://www.prnewswire.com/news-releases/roivant-unveils-targeted-protein-degradation-platform-301186928.html

Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch “Vants” – nimble and focused biopharmaceutical and health technology companies. These Vants include Proteovant but also Dermovant, ImmunoVant,as well as others.

Roivant’s drug discovery capabilities include the leading computational physics-based platform for in silico drug design and optimization as well as machine learning-based models for protein degradation.

The integration of our computational and experimental engines enables the rapid design of molecules with high precision and fidelity to address challenging targets for diseases with high unmet need.

Our current modalities include small molecules, heterobifunctionals and molecular glues.

Roivant Unveils Targeted Protein Degradation Platform

– First therapeutic candidate on track to enter clinical studies in 2021

– Computationally-designed degraders for six targets currently in preclinical development

– Acquisition of Oncopia Therapeutics and research collaboration with lab of Dr. Shaomeng Wang at the University of Michigan to add diverse pipeline of current and future compounds

Clinical-stage degraders will provide foundation for multiple new Vants in distinct disease areas

– Platform supported by $200 million strategic investment from SK Holdings

Other articles in this Vibrant Philly Biotech Scene on this Online Open Access Journal include:

The Vibrant Philly Biotech Scene: PCCI Meeting Announcement, BioDetego Presents Colon Cancer Diagnostic Tool

The Vibrant Philly Biotech Scene: Focus on KannaLife Sciences and the Discipline and Potential of Pharmacognosy

The Vibrant Philly Biotech Scene: Focus on Vaccines and Philimmune, LLC

The Vibrant Philly Biotech Scene: Focus on Computer-Aided Drug Design and Gfree Bio, LLC

Philly Biotech Scene: Biobots and 3D BioPrinting (Now called Allevi)

Philly Biotech Scene: November 2015 PCCI Meeting Showcasing ViFant (Penn Center For Innovation)

Spark Therapeutics’ $4.8Billion deal Confirmed as Biggest VC-backed Exit in Philadelphia

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Tweets and Re-Tweets of Tweets by @pharma_BI@AVIVA1950 at 2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

REAL TIME EVENT COVERAGE as PRESS by invitation from 2021 Virtual World Medical Innovation Forum at #WMIF2021 @MGBInnovation:

Aviva Lev-Ari, PhD, RN

Tweet Collection Curator:

Aviva Lev-Ari, PhD, RN

UPDATED Twitter Analytics

May 2021  31 days

TWEET HIGHLIGHTS

Top Tweet earned 611 impressions

@MGBInnovation#WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950@pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included pic.twitter.com/9Q2dWikhNd 1  2

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Ryan Gravatt@gravatt FOLLOWS YOU

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Top mention earned 15 engagements

#COVID#vaccines by @Pfizer, @AstraZeneca are probed in @Europe after reports of #heart#inflammation, rare #nerve#disorderpharmaceuticalintelligence.com/2021/05/14/cov… via @pharma_BI@AVIVA1950 1  3View all Tweet activityView Tweet activity

MAY 2021 SUMMARY

Tweets

213

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17.6K

Profile visits

861

Mentions

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These are the Tweets and the Re-Tweets

by Day, 5/21, 5/20, 5/19 for

2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

Real Time coverage: Aviva Lev-Ari, PhD, RN

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Aviva Lev-Ari, PhD, RN, Founder, 1.0 LPBI Group and 2.0 LPBI Group

May 21, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 21, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 21, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

From: Mass General Brigham <innovations@partners.org>
Reply-To: <innovations@partners.org>
Date: Monday, May 24, 2021 at 9:31 AM
To: “Aviva Lev-Ari, PhD, RN” <AvivaLev-Ari@alum.berkeley.edu>
Subject: RECAP | World Forum | Day 3 | GCT | CEOs | Harvard | Investors

Notable Tweets
@mandywoodland Fascinating #WMIF2021 panel on mRNA yesterday -“mRNA is the message, and we just have to decide what message we want to deliver to the cell,” said moderator Lindsey Baden, MD. “The promise of this technology could not be more front and center for all of us.”   @LeapsByBayer Congratulations to the 2021 Innovation Discovery Grants winners: @lynchielydia, Peter Sage, @GrishchukL, Benjamin Kleinstiver, Petr Baranov, announced at the #WMIF2021. It’s exciting to see the range of breakthrough research in #geneticdisease at @MassGenBrigham@DrLilitGaribyan Gene and cell therapy have scalability problems that we need to solve. This is what is echoed this week at @MGBInnovation World Medical Innovation Forum. #gct #celltherapy #healthcare #innovation   @MPDexpert “imagine how the future could look if gene therapy cost 1/100th what it does today” @VCAmir @PolarisVC #wmif2021  
@AVIVA1950 #WMIF2021 @MGBInnovation Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT Multi OMICS and academia originated innovations are the most attractive areas @pharma_BI @AVIVA1950
Notable Tweets

 

Disruptive Dozen

2021 World Medical Innovation Forum on

YouTube

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Aviva Lev-Ari

@AVIVA1950

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#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

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Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

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 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

 PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 21, 2021

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Erwan Bezard, PhD INSERM Research Director, Institute of Neurodegenerative Diseases Cautious on reversal

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Nikola Kojic, PhD CEO and Co-Founder, Oryon Cell Therapies Autologus cell therapy placed focal replacing missing synapses reestablishment of neural circutary

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Bob Carter, MD, PhD Chairman, Department of Neurosurgery, MGH William and Elizabeth Sweet, Professor of Neurosurgery, HMS Neurogeneration REVERSAL or slowing down? 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS efficacy Autologous cell therapy transplantation approach program T cells into dopamine genetating cells greater than Allogeneic cell transplantation 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS Pharmacologic agent in existing cause another disorders locomo-movement related 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT Multi OMICS and academia originated innovations are the most attractive areas

@pharma_BI

@AVIVA1950

1

3

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT 

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Oleg Nodelman Founder & Managing Partner, EcoR1 Capital Invest in company next round of investment will be IPO 20% discount

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Peter Kolchinsky, PhD Founder and Managing Partner, RA Capital Management Future proof for new comers disruptors  Ex Vivo gene therapy to improve funding products what tool kit belongs to 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

Deep Nishar Senior Managing Partner, SoftBank Investment Advisors Young field vs CGT started in the 80s  high payloads is a challenge 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Bob Carter, MD, PhD MGH, HMS cells producing dopamine transplantation fibroblast cells metabolic driven process lower mutation burden  Quercetin inhibition elimination undifferentiated cells graft survival oxygenation increased 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Chairman, Department of Neurosurgery, MGH, Professor of Neurosurgery, HMS Cell therapy for Parkinson to replace dopamine producing cells lost ability to produce dopamine skin cell to become autologous cells reprogramed  

@pharma_BI

@AVIVA1950

#WMIF2021

@MGBInnovation

Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH Off-th-shelf one time treatment becoming cure  Intact tissue in a dish is fragile to maintain metabolism to become like semiconductors

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Ole Isacson, MD, PhD Director, Neuroregeneration Research Institute, McLean Professor, Neurology and Neuroscience, MGH, HMS Opportunities in the next generation of the tactical level Welcome the oprimism and energy level of all

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Erin Kimbrel, PhD Executive Director, Regenerative Medicine, Astellas In the ocular space immunogenecity regulatory communication use gene editing for immunogenecity Cas1 and Cas2 autologous cells

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Nabiha Saklayen, PhD CEO and Co-Founder, Cellino scale production of autologous cells foundry using semiconductor process in building cassettes by optic physicists

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Joe Burns, PhD VP, Head of Biology, Decibel Therapeutics Ear inside the scall compartments and receptors responsible for hearing highly differentiated tall ask to identify cell for anticipated differentiation control by genomics

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH first drug required to establish the process for that innovations design of animal studies not done before 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Meredith Fisher, PhD Partner, Mass General Brigham Innovation Fund Strategies, success what changes are needed in the drug discovery process@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Manufacturing change is not a new clinical trial FDA need to be presented with new rethinking for big innovations Drug pricing cheaper requires systematization

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

Kush Parmar, MD, PhD Managing Partner, 5AM Ventures Responsibility mismatch should be and what is “are”

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

5h

#WMIF2021

@MGBInnovation

David Berry, MD, PhD CEO, Valo Health GP, Flagship Pioneering Bring disruptive frontier platform reliable delivery CGT double knockout disease cure all change efficiency scope human centric vs mice centered right scale acceleration

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

6h

#WMIF2021

@MGBInnovation

Kush Parmar, MD, PhD Managing Partner, 5AM Ventures build it yourself, benefit for patients FIrst Look at MGB shows MEE innovation on inner ear worthy investment  

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

6h

#WMIF2021

@MGBInnovation

Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Frustration with supply chain during the Pandemic, GMC anticipation in advance CGT rapidly prototype rethink and invest proactive investor .edu and Pharma

@pharma_BI

@AVIVA1950

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 21, 2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

The # of US patients with Parkinson’s Disease is expected to double over next 30 years. Penelope Hallett PhD, Co-Director of the Neuroregeneration Research Inst

@McLeanHospital

, presents a #regenerativemedicine approach that could alter that trajectory. #WMIF2021

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Our “Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing” panelists have taken the stage. #WMIF2021

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

CAR-T therapies have proven remarkably effective. Now,

@MassGenBrigham

researchers including

@MGHCancerCenter

Marcela Maus, MD PhD, are working to expand the reach of this transformative technology. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #9. Building the Next Wave of CAR-T-cell Therapies #WMIF2021 #GCT #GeneAndCellTherapy #CellTherapy #CarT #DisruptiveDozen

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Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #6. Eyes and Ears: Expanding Gene Therapy’s Reach #WMIF2021 #GCT #GeneAndCellTherapy #GeneTherapy #DisruptiveDozen

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 20

If you’ve missed some First Look sessions, don’t worry! We’ve got you covered. Our First Look On-Demand videos, featuring 18

@MassGenBrigham

investigators giving previews of their #GCT research, are available to view on the #WMIF2021 conference platform. https://worldmedicalinnovation.org/register/

5

7

You Retweeted

REGENXBIO

@REGENXBIO

·

May 19

This morning at 10:20 a.m. ET, our CEO, Ken Mills, will be participating live on the AAV Success Studies virtual panel at the #WMIF2021, hosted by

@MGBInnovation

. Click here to register: https://bit.ly/33tHTti #Genetherapy

Register | World Medical Innovation Forum – Gene and Cell Therapy

Hear from industry-leading experts discuss the advances and future of GCT in health care. May 19-21, 2021; Mass General Brigham. Register!

worldmedicalinnovation.org

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You Retweeted

Brett P. Monia, Ph.D.

@BPMonia

·

May 20

Looking forward to joining

@MGBInnovation

and global colleagues at #WMIF2021. On Thursday, May 20, my colleagues and I will discuss the advantages of RNA-targeted medicines and how they might shape the future of medicine for patients.

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · May 10

Are you part of the @MassGenBrigham network and interested in #GeneAndCellTherapy? Join us at the World Medical Innovation Forum on 5/19-5/21. Register today! https://worldmedicalinnovation.org/register/ #WMIF2021

1

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You Retweeted

Maria Luiza Gutierrez de Andrade Seixas

@MLGASeixas

·

May 16

Incredible opportunity to get up to speed with the most innovative technologies in medicine ! Gene and cell therapy are revolutionizing healthcare ! #WMIF2021 #MedTwitter

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · May 11

#WMIF2021 is an opportunity for innovators from around the globe to meet, explore, challenge, and reflect on the issues influencing the adoption of novel technologies in #healthcare. Register now to join the conversation: https://worldmedicalinnovation.org/register/

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Currently, the only cure for some common blood disorders is a bone marrow transplant, which can be risky. Now, gene therapies are also in the works, including a CRISPR-based #genetherapy being tested in clinical trials with encouraging early results. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #2. A Genetic Fix for Two Common Blood Disorders #WMIF2021 #GCT #GeneAndCellTherapy #BloodDisorders #DisruptiveDozen

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Researchers have pinpointed key genes involved in cholesterol and lipid metabolism that represent promising targets for new cholesterol-lowering treatments. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #1. A New Generation of Cholesterol-Loweing Therapies #WMIF2021 #GCT #GeneAndCellTherapy #DisruptiveDozen

2

1

You Retweeted

Harvard Ophthalmology

@HMSeye

·

May 19

The

@MGBInnovation

#WMIF2021 event kicks of this morning! Congratulations to faculty member and event Co-Chair

@VandenbergheLuk

on putting together such a terrific program. Register: https://bit.ly/3uWYB0E

4

9

You Retweeted

Yulia Grishchuk Lab

@GrishchukL

·

4h

I really enjoyed this remarkable panel #WMIF2021. Thank you Meredith Fisher for moderating and thank you David, Bob and Kush for openly sharing your big picture view

1

4

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Thank you to our World Medical Innovation Forum Collaborators

@Amplify_Bio

@bostonsci

@CanonUSA

@CatalentPharma

@InterSystems

@nlvcofficial

@onemedical

@ReconStrategy

@SiemensHealth

@thermofisher

@VertexPharma

#WMIF2021

You Retweeted

Tracy Doyle

@doylet

·

5h

Variability, delays, manufacturing as an afterthought make #GCT challenging from an investment POV — need to rethink the ecosystem and drive efficiency, invest in tech innovation says Bob Nelson ARCH Venture Partners

@MGBInnovation

#WMIF2021

1

You Retweeted

Tracy Doyle

@doylet

·

5h

We need to change the scale and scope of how #GCT is advancing from discovery to development — systematization critical. Can’t have thousands of one-off therapies say early-stage investors. Major mis-match between where things are now and what could be.

@MGBInnovation

#WMIF202

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #8. Replacing What’s Lost: Stem Cell Therapies for Diabetes #WMIF2021 #GCT #GeneAndCellTherapy #StemCell #StemCellResearch #Diabetes #DisruptiveDozen

3

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

21h

An overview of our CEO Panel featuring Lisa Deschamps of

@NovartisGene

, Kieran Murphy of

@GEHealthcare

and Christian Rommel PhD, of

@Bayer

#WMIF2021

4

7

You Retweeted

Mass General Brigham

@MassGenBrigham

·

4h

Gene and cell therapies could change the future of medicine for patients w chronic disease or rare/ultra-rare disease – hear how

@MassGenBrigham

is working w the GCT ecosystem to drive new discoveries from bench to bedside #GCT #WMIF2021

5

11

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

That’s a wrap! Thank you to everyone who helped make #WMIF2021 such a success, especially our incredible sponsors:

@NovartisGene

@Bayer

@GEHealthcare

@AstellasUS

@biogen

@FujifilmHealth

and more. Full list: https://worldmedicalinnovation.org/sponsors/

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #1. A New Generation of Cholesterol-Loweing Therapies #WMIF2021 #GCT #GeneAndCellTherapy #DisruptiveDozen

5

2

You Retweeted

Natalie Artzi

@NatalieArtzi

·

17h

Today I moderated a panel on Gene and Cell Therapy Delivery, Perfecting the Technology. We highlighted non-viral delivery technologies as key enablers of gene therapy and editing. Learn more: https://lnkd.in/d-Xqzqh #WMIF2021

3

12

You Retweeted

Yulia Grishchuk Lab

@GrishchukL

·

5h

Thank you

@MGBInnovation

and

@LeapsByBayer

for this award! Congratulations to

@BKleinstiver

and all other winners!

@MGH_RI

@CGM_MGH

! #WMIF2021

Quote Tweet

Leaps by Bayer

@LeapsByBayer

 · 6h

Congratulations to the 2021 Innovation Discovery Grants winners: @lynchielydia, Peter Sage, @GrishchukL, Benjamin Kleinstiver, Petr Baranov, announced at the #WMIF2021. It’s exciting to see the range of breakthrough research in #geneticdisease at @MassGenBrigham…

Show this thread

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Natalie Artzi

@NatalieArtzi

·

17h

An artistic description of an exciting panel I led today, at the World Biomedical Innovation Forum, discussing the future of non-viral delivery systems for gene therapy. #MatthewStanton #LauraSeppLorenzino #DouglasWilliams #SonyaMontgomery #WMIF2021

May 20, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 20, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 20, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

Example for a TWEET

Aviva Lev-Ari

@AVIVA1950

·

May 21

@MGBInnovation

#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

@pharma_BI

Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

2

Example for a RE-TWEET

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 21

Thanks

@AVIVA1950

for sharing this screen capture of the impressive lineup of #GCT “Disruptive Dozen” panelists at #WMIF2021

Quote Tweet

Aviva Lev-Ari

@AVIVA1950

 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 20, 2021

Aviva Lev-Ari

@AVIVA1950

·

2h

#WMIF2021

@MGBInnovation

Bob Brown, PhD CSO, EVP of R&D, Dicerna small molecule vs capacity of nanoparticles to deliver therapeutics quantity for more molecule is much larger CNS delivery most difficult

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Jeannie Lee, MD, PhD Molecular Biologist, MGH Prof Genetics, HMS 200 disease X chromosome unlock for neurological genetic diseases: Rett Syndrome, autism spectrum disorders female model vs male mice model restore own protein

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Suneet Varma Global President of Rare Disease, Pfizer review of protocols and CGT for Hemophilia Pfizer: You can’t buy Time With MIT Pfizer is developing a model for Hemophilia CGT treatment

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Gallia Levy, MD, PhD CMO, Spark Therapeutics Hemophilia CGT is the highest potential for Global access logistics in underdev countries working with NGOs practicality of the Tx Roche reached 120 Counties great to be part of the Roche

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Theresa Heggie CEO, Freeline Therapeutics Safety concerns, high burden of treatment CGT has record of safety and risk/benefit adoption of Tx functional cure CGT is potent Tx relative small quantity of protein needs be delivered 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Suneet Varma Global President of Rare Disease, Pfizer Gene therapy at Pfizer small, large molecule and CGT – spectrum of choice allowing Hemophilia patients to marry 1/3 internal 1/3 partnership 1/3 acquisitions  review of protocols

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio What strain of Flu vaccine will come back in the future when people do not use masks. AAV vectors small transcript size fit reach cytoplasm more development coming

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna Flu vaccine knowing the virus variant 45 days for Personalized cancer vaccine one per patient

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna Many years of mRNA pivoting for new diseases, DARPA, nucleic Acids global deployment of a manufacturing unit on site where the need arise Elan Musk funds new directions at Moderna

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy and the 

@pharma_BI

@AVIVA1950

1

2

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Lindsey Baden, MD Director, Clinical Research, Division of Infectious Diseases, BWH Associate Professor, HMS In vivo delivery process regulatory for new opportunities for same platform new indication using multi valence vaccines

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech

@pharma_BI

@AVIVA1950

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna Many years of mRNA pivoting for new diseases, DARPA, nucleic Acids global deployment of a manufacturing unit on site where the need arise Elan Musk funds new directions at Moderna

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio 1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Kate Bingham, UK Vaccine Taskforce July 2020, AAV vs mRNA delivery across UK local centers administered both types supply and delivery uplift 

@pharma_BI

@AVIVA1950

1

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Melissa Moore CSO, Moderna mRNA vaccine 98% efficacy for Pfizer and Moderna more then 10 years 2015 mRNA was ready (ZIKA, RSV), as the proteine is identify manufacturing temp less of downside in the future ability to store at Ref

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Shunfei Yan, PhD Investment Manager, InnoStar Capital Indication driven: Hymophilia,  Allogogenic efficiency therapies Licensing opportunities 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Richard Wang, PhD CEO, Fosun Kite Biotechnology Co. Ltd Possibilities to be creative and capitalize the new technologies for new drug Support of the ecosystem by funding new companies Autologous in patients differences cost challenge

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Tian Xu, PhD Vice President, Westlake University ICH Chinese FDA -r regulation similar to the US Difference is the population recruitment, in China patients are active participants Dev of transposome non-viral methods, price

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Alvin Luk, PhD CEO, Neuropath Therapeutics Monogenic rare disease with clear genomic target Increase of 30% in patient enrollment  Regulatory reform approval is 60 days no delay

@pharma_BI

@AVIVA1950

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 20, 2021

You Retweeted

Vertex Pharmaceuticals

@VertexPharma

·

May 19

We’re excited to attend this week’s #WMIF2021 to talk all things cell and genetic therapies. Join our Chief of VCGT Bastiano Sanna tomorrow at 9:50am EDT for a discussion on the promise of cell therapies for type 1 diabetes. Register now! https://bit.ly/3otngYd

2

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

8h

John Fish, Board Chair, Brigham Health, Chairman & CEO, Suffolk on the Novartis Main Stage to introduce the “Collaboration is Key: GCT R&D of the Future” fireside chat with Jay Bradner, MD, President, NIBR

@NovartisScience

. #WMIF2021

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

In our next First Look presentation we’ll hear from Xandra Breakefield PhD & Koen Breyne PhD

@MGHNeurology

@MGHNeurosurg

about their work focused on developing non-viral vectors to enhance #genedelivery. #WMIF2021 #GCT #genetherapy

More Topics

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 19

Thomas VanCott, PhD, Chief Technology & Strategy Officer, Catalent Cell & Gene Therapy, says that time, improvements and scaling up in manufacturing will lead to allogeneic cell therapies. He recognizes that upfront costs are high, but will decrease in the long term #WMIF2021

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Ravi Thadhani, CAO

@MassGenBrigham

and Juergen Eckhardt, Head of

@LeapsbyBayer

, are announcing the 2021 Innovation Discovery Grants this afternoon at #WMIF2021.

1

3

You Retweeted

Editas Medicine

@editasmed

·

10h

Today Lisa Michaels, Editas CMO, will participate in the panel “Gene Editing – Achieving Therapeutic Mainstream” at the World Medical Innovation Forum #WMIF2021 in Boston. For those attending, be sure to tune in!

@MassGenBrigham

https://bit.ly/3hx1XTV #geneediting #biotechnology

Gene Editing | Achieving Therapeutic Mainstream – 2021 World Medical Innovation Forum

Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify…

worldmedicalinnovation.org

1

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

A behind the scenes peek at our “Gene Editing | Achieving Therapeutic Mainstream” moderator & panelists preparing to go live. #WMIF2021

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Thank you to the “Common Blood Disorders | Gene Therapy” moderator David Scadden, MD

@ScaddenLab

@harvardstemcell

and panelists Leslie Kean, MD PhD

@DanaFarberNews

, Samarth Kulkarni, PhD

@CRISPRTX

, Nick Leschly

@bluebirdbio

, Mike McCune, MD PhD

@gatesfoundation

. #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Kieran Murphy, CEO,

@GEHealthcare

, views GCT as the ultimate precision medicine. AI, machine learning, and data science comprise one of the big disruptive forces that will address misdiagnosis, smooth out workflow, reduce cost and enhance recovery. #WMIF2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Wrapping up Day 2 of #WMIF2021 with the “Gene Expression | Modulating with Oligonucleotide-Based Therapies” panel.

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Juergen Eckhardt, Head of

@LeapsbyBayer

, announces new Bayer mentoring program for Innovation Discovery Grant winners at #WMIF2021.

3

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

In our final First Look session of the day, Pierpaolo Peruzzi, MD PhD,

@BWHNeurosurgery

presents “RNA Therapy for Brain Cancer” #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Hear from

@intelliatweets

CSO Laura Sepp-Lorenzino, PhD, in our “GCT Delivery | Perfecting the Technology” panel this afternoon! #WMIF2021

Quote Tweet

Intellia Therapeutics

@intelliatweets

 · 6h

Today, Intellia CSO, @LauraSeppLore will be participating in the World Medical Innovation Forum’s panel on Gene and Cell Therapy Delivery, Perfecting the Technology. #WMIF2021 @MGBInnovation. Click here to learn more: https://worldmedicalinnovation.org

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Natalie Artzi, PhD, Assistant Professor

@BrighamWomens

is back with us this afternoon sharing a First Look at “Versatile Polymer-Based Nanocarriers for Targeted Therapy and Immunomodulation.” #WMIF2021 #GCT #geneandcelltherapy

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

8h

We look forward to hearing from

@ViaCyte

VP of Clinical Development, Manasi Jaiman, during the “Diabetes | Grand Challenge” panel today. #WMIF2021

Quote Tweet

ViaCyte

@ViaCyte

 · 8h

Join us at #WMIF2021 today! Our own Manasi Jaiman, VP, Clinical Development, will participate in the Diabetes: Grand Challenge panel to discuss regenerative medicine approaches for T1D utilizing stem-cell derived islet cell replacement therapy.

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

5h

We’ll see you back here after the break for the “GCT Delivery | Perfecting the Technology” panel, featuring moderator Natalie Artzi, PhD,

@BrighamWomens

and panelists from

@EvOx_Ltd

,

@intelliatweets

,

@generationbio

and

@codiakbio

. #WMIF2021

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Christian Rommel, PhD,EVP, Head, Pharmaceuticals Research & Development,

@Bayer

, discusses how GCT is in the embryonic phase. Bayer is ready to treat its first Parkinson’s patient, and is exploring therapeutic technologies to treat diseases with single gene defects #WMIF2021

1

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Mass General Brigham Innovation

@MGBInnovation

·

8h

Next up is the #Diabetes | Grand Challenge panel at #WMIF2021 featuring speakers from

@BrighamWomens

@armi_usa

@ViaCyte

@VertexPharma

@Sigilon_Inc

3

5

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

10h

The “Gene Editing | Achieving Therapeutic Mainstream” panel today at 2:55 pm Eastern will discuss the movement of #geneediting technology into the therapeutic mainstream. Join us! #WMIF2021 https://worldmedicalinnovation.org/register/

Quote Tweet

Editas Medicine

@editasmed

 · 10h

Today Lisa Michaels, Editas CMO, will participate in the panel “Gene Editing – Achieving Therapeutic Mainstream” at the World Medical Innovation Forum #WMIF2021 in Boston. For those attending, be sure to tune in! @MassGenBrigham https://bit.ly/3hx1XTV #geneediting #biotechnology

You Retweeted

Atara Bio

@Atarabio

·

2h

Global Head of R&D

@jdupontmd

joined this week’s World Medical Innovation Forum hosted by

@MGBInnovation

to discuss the current state of CAR-T and its future prospects. These conversations are important for the development of potential #CART therapies. #WMIF2021

1

8

You Retweeted

Tracy Doyle

@doylet

·

9h

“We can get to an “n of 1” with mRNA technology says Melissa Moore, PhD, CSO Platform Research,

@moderna_tx

@MGBInnovation

#WMIF2021 #GCT

1

1

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Intellia Therapeutics

@intelliatweets

·

6h

Today, Intellia CSO,

@LauraSeppLore

will be participating in the World Medical Innovation Forum’s panel on Gene and Cell Therapy Delivery, Perfecting the Technology. #WMIF2021

@MGBInnovation

. Click here to learn more: https://worldmedicalinnovation.org

3

4

You Retweeted

TranslateBio

@TranslateBio

·

7h

Graphical representation of this morning’s #mRNA #vaccines panel at

@MGBInnovation

‘s #WMIF2021 — Thanks to the MGB team for facilitating a great discussion!

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 7h

Overview of our #mRNA Vaccines panel today, highlighting improved manufacturing capabilities & potential for #personalizedmedicine. Thank you to Lindsey Baden @bwh_id & panelists Kate Bingham, SV Health Investors, Melissa Moore @moderna_tx and Ron Renaud @TranslateBio #WMIF2021

1

3

You Retweeted

Tracy Doyle

@doylet

·

May 19

18

@MassGenBrigham

investigators are ready to give you an early preview of their #GCT research in the First Look sessions at #WMIF2021. Exciting opportunities to dramatically change how disease is treated!

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

7h

Please welcome Marc Casper, CEO

@thermofisher

to the stage for a Fireside Chat moderated by Erin Harris

@ErinHarris_1

, Editor in Chief

@_CellandGene

“Partnering Across the GCT Spectrum” #WMIF2021 #GCT #geneandcelltherapy

4

3

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

The “CEO Panel | Anticipating Disruption | Planning for Widespread GCT” panelists have joined the stage. #WMIF2021

1

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Mass General Brigham Innovation

@MGBInnovation

·

7h

Our “Rare and Ultra Rare Diseases | GCT Breaks Through” panelists on the role of family organizations & patient advocacy groups in moving us forward on the regulatory side – “It’s absolutely essential” #WMIF2021

2

1

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Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Lydia Lynch PhD, Brigham and Women’s Hospital receives an Innovation Discovery Grant for “Generating Superior ‘Killers’ for Adoptive Cell Therapy in Cancer” at #WMIF2021.

@BrighamWomens

@BrighamResearch

2

You Retweeted

Tracy Doyle

@doylet

·

10h

Looking forward to the Diabetes Grand Challenge and how #GCT could help millions of people. Read about what facing this disease and how cell therapies could lessen the burden from Manasi Jaiman, MD, VP, Clinical Development

@ViaCyte

here http://bit.ly/T1Dcelltherapies… #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 11h

Today is Day 2 of the World Medical Innovation Forum. Which panel you are most excited to see today? Reply and let us know! #WMIF2021 https://worldmedicalinnovation.org/agenda/

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Coming up at 12:05 pm Eastern: “CEO Panel | Anticipating Disruption | Planning for Widespread GCT” featuring panelists from

@NovartisGene

@GEHealthcare

@Bayer

and moderated by

@CNBC

Senior Health and Science Reporter

@megtirrell

#WMIF2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

10h

Join us at #WMIF2021 to hear from Suneet Varma, Global President of Rare Disease

@Pfizer

, during the “Benign Blood Disorders” today at 9:00 am Eastern. https://worldmedicalinnovation.org/register/

Quote Tweet

Pfizer Inc.

@pfizer

 · May 19

Cell and gene therapies hold promising potential for rare disease, blood cancers, and viral diseases. Register for #WMIF21 to hear about our work to pioneer cutting-edge science across our pipeline to advance breakthroughs that change patients’ lives: https://on.pfizer.com/3f3CGzj

2

1

You Retweeted

Pearl Freier

@PearlF

·

9h

Melissa Moore/Moderna said they are working with Merck on developing personalized cancer vaccines, n of 1 #wmif2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Peter Sage PhD, Brigham and Women’s Hospital receives an Innovation Discovery Grant for “Novel Strategies to Enhance Tfr Treatment of Autoimmunity” at #WMIF2021

@BrighamWomens

@BrighamResearch

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Yulia Grishchuk PhD, Massachusetts General Hospital, receives an Innovation Discovery Grant for “AAV-Based Gene Replacement Therapy Improves Targeting and Clinical Outcomes in a Childhood CNS Disorder” at #WMIF2021

@MassGeneralNews

@MGH_RI

@CGM_MGH

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Jinjun Shi, PhD, Brigham and Women’s Hospital, receives an Innovation Discovery Grant for “Long-Lasting mRNA Therapy for Genetic Disorders” at #WMIF2021

@BrighamWomens

@BrighamResearch

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

9h

Final thoughts from “Benign Blood Disorders” panelists on academic/industry collaboration — the pace of #innovation is incredibly exciting, and I think it will be even faster together. #WMIF2021

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Benjamin Kleinstiver PhD, Massachusetts General Hospital, receives an Innovation Discovery Grant for “Towards a Permanent Genetic Cure for Spinal Muscular Atrophy” at #WMIF2021

@MassGeneralNews

@MGH_RI

@CGM_MGH

2

You Retweeted

Pearl Freier

@PearlF

·

10h

Listening to mRNA vaccines #wmif2021 panel w/ speakers incl SV Health managing partner & ex UK Vaccine Taskforce

@katebingham2

, Moderna CSO Platform Rsrch Melissa Moore,

@TranslateBio

CEO Ron Renaud

@biotech1969

, Brigham/BWH Dir Clinical Research Infectious Disease Lindsey Baden

2

2

You Retweeted

Ned Pagliarulo

@NedPagliarulo

·

May 19

FDA’s Peter Marks, at #WMIF2021, notes # of INDs for gene therapies was flat in 2020 vs. 2019. But the fact IND submissions didn’t decline, he said, is a sign of how strong the gene therapy field is, given pandemic’s disruption.

1

9

21



You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

5h

Coming up this afternoon: the 2021 Innovation Discovery Grants in #geneandcelltherapy. Who will secure additional funding for research to advance #GCT? Join us to watch live. #WMIF2021 https://worldmedicalinnovation.org/register/

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

1h

Thank you Jeannie Lee, MD PhD

@MGHPathology

, Bob Brown, PhD

@DicernaPharma

, Brett Monia, PhD

@ionispharma

, and Alfred Sandrock, MD PhD

@biogen

for sharing your perspectives on oligonucleotide-based therapies. #WMIF2021

1

2

You Retweeted

Pearl Freier

@PearlF

·

9h

Melissa Moore/Moderna- one advantage of mRNA is ability to do multivalent vaccines she said. She said they are already testing multivalent covid vaccines in clinical trials & testing flu vaccines. #wmif2021

1

3

You Retweeted

Pearl Freier

@PearlF

·

10h

Kate Bingham/SV Health & former head of UK Vaccine Taskforce: they haven’t seen escape variants in UK yet she said. mRNA is quickest platform to address escape variants probably. Needle delivery w/ supply cold chain has been the challenge. Deploying 3 vaccines in UK #WMIF2021

1

1

1

Show this thread

You Retweeted

Tracy Doyle

@doylet

·

6h

Marc Casper

@thermofisher

says gene and cell therapy represents a “phenomenal opportunity to improve patients’ lives” #WMIF2021 #GCT

1

2

You Retweeted

TranslateBio

@TranslateBio

·

7h

Today, our CEO Ron Renaud

@biotech1969

participated in

@MGBInnovation

‘s 2021 World Medical Innovation Forum to discuss the impact of #messengerRNA #vaccines on the industry #WMIF2021 #mRNA

2

10

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Lisa Deschamps, SVP & Chief Business Officer,

@NovartisGene

, notes that the science behind gene cell therapies is converging with technological development. How therapies are brought to market is still the question, as there is no roadmap when reimagining medicine #WMIF2021

3

4

You Retweeted

Pearl Freier

@PearlF

·

10h

Melissa Moore/Moderna: clear advantage of mRNA vaccine is how quickly we can manufacture the vaccines. Downsides- need 2store at low temperatures & limited shelflife 4storage in refrigerator. I know that both companies [Moderna, Pfizer/BioNTech] r working 2change this #wmif2021

You Retweeted

Novartis Gene Therapies

@NovartisGene

·

6h

We’re committed to addressing the unmet needs of people living with rare genetic diseases. Our SVP, External Innovation and Strategic Alliances, Leah Bloom, discusses the promise #genetherapy holds for communities impacted by rare diseases during #WMIF2021.

2

4

You Retweeted

Tracy Doyle

@doylet

·

6h

Diagnostics and data tools key part of precision medicine complementing gene and cell therapy says

@KieranMurphyCEO

@GEHealthcare

at

@MGBInnovation

#WMIF2021

Meg Tirrell and 2 others

2

2

You Retweeted

Tracy Doyle

@doylet

·

7h

Debating the value of natural history studies in rare/ultra rare disease — panel led by Susan Slaugenhaupt, PhD, scientific director,

@MGH_RI

at #WMIF2021. Challenges include costs, feasibility, timing, comparative data.

1

2

You Retweeted

Tracy Doyle

@doylet

·

8h

Rett’s Syndrome, which primarily affects young girls, has historically been studied in male mice! Jeannie Lee, MD, PhD,

@MassGeneralNews

, and team are exploring how to treat the disease w X chromosome reactivation… and using a female mouse model. Hear more on #GCT at #WMIF2021

2

5

You Retweeted

Tracy Doyle

@doylet

·

10h

Speed of vaccination is critical to prevent escape variants says Kate Bingham, SV Health Investors, UK, at #WMIF2021, exploring what’s next for the technology w panel led by Lindsey Baden MD,

@BrighamWomens

2

2

May 19, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 19, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 19, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

Example for a TWEET

Aviva Lev-Ari

@AVIVA1950

·

May 21

@MGBInnovation

#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

@pharma_BI

Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

2

Example for a RE-TWEET

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 21

Thanks

@AVIVA1950

for sharing this screen capture of the impressive lineup of #GCT “Disruptive Dozen” panelists at #WMIF2021

Quote Tweet

Aviva Lev-Ari

@AVIVA1950

 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

 PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 19, 2021



Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Marcela Maus, MD, PhD Director, Cancer Center, MGH, HMS  Fit-to-purpose CAR-T cells: 3 lead programs Tr-fill CAR-T induce response myeloma and multiple myeloma GBM 27 patents on CAR-T +400 patients treaded 40 Clinical Trials 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Thomas VanCott, PhD Global Head of Product Dev, Gene & Cell Therapy, Catalent 2/3 autologous 1/3 allogeneic  CAR-T high doses scale up is not done today logistics issues centralized vs decentralized allogeneic are health donors

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Ropa Pike, Director,  Enterprise Science & Partnerships, Thermo FIsher Scientific  Centralized biopharma industry is moving  to decentralized models site specific license 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Rahul Singhvi, ScD CEO and Co-Founder, National Resilience, Inc. Investment company in platforms to be shared by start ups in CGT. Production cost of allogeneic: cost of quality 30% reagents 30% cell 30% Test is very expensive 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Oladapo Yeku, MD, PhD Clinical Assistant in Medicine, MGH Outstanding moderator and most gifted panel on solid tumor success window of opportunities studies 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Knut Niss, PhD CTO, Mustang Bio tumor hot start in 12 month clinical trial solid tumors Combination therapy will be an experimental treatment long journey checkpoint inhibitors to be used in combination maintenance 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Barbra Sasu, PhD CSO, Allogene T cell response at prostate cancer  tumor specific  cytokine tumor specific signals move from solid to metastatic cell type for easier infiltration

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Jennifer Brogdon Executive Director, Head of Cell Therapy Research, Exploratory Immuno-Oncology, NIBR 2017 CAR-T first approval M&A and research collaborations TCR tumor specific antigens avoid tissue toxicity 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Jay Short, PhD Chairman, CEO, Cofounder, BioAlta, Inc. Tumor type is not enough for R&D therapeutics other organs are involved in periphery difficult to penetrate solid tumors biologics activated in the tumor only, positive changes

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Christi Shaw CEO, Kite CAR-T is priority 120 companies in the space Manufacturing consistency  Patients respond with better quality of life

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Stefan Hendriks Global Head, Cell & Gene, Novartis Confirmation the effectiveness of CAR-T therapies, 1 year response to 5 years 26 months Patient not responding a lot to learn Patient after 8 months of chemo can be helped by CAR-T

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Jeffrey Infante, MD , Oncology, Janssen R&D Direct effect with intra-tumor single injection with right payload Platform approach  Prime with 1 and Boost with 2 – not yet experimented with  Do not have the data at trial

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Nino Chiocca, MD, PhD Neurosurgeon-in-Chief BWH, HMS Oncolytic therapy DID NOT WORK Pancreatic Cancer and Glioblastoma Intra-tumoral heterogeniety hinders success Oncolytic VIRUSES – “coldness” GADD-34 20,000 GBM 40,000 pancreatic

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Loic Vincent, PhD Head of Oncology Drug Discovery Unit, Takeda Classification of Patients by prospective response type id UNKNOWN yet, population of patients require stratification

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Loic Vincent, PhD Head of Oncology Drug Discovery Unit, Takeda R&D in collaboration with Academic Vaccine platform to explore different payload IV administration may not bring sufficient concentration to the tumor is administer IV

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Nino Chiocca, MD, PhD Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH Harvey W. Cushing Professor of Neurosurgery, HMS Challenges of manufacturing at Amgen what are they?

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

David Reese, MD Executive Vice President, R&D , Amgen Inter lesion injection of agent vs systemic therapeutics cold tumors immune resistant render them immune susptible Oncolytic virus is a Mono therapy addressing the unknown 

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

David Reese, MD Executive Vice President, Research and Development, Amgen Inter lesion injection of agent vs systemic therapeutics  cold tumors immune resistant render them immune suseptible Oncolytic virus is a Mono therapy

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Robert Coffin, PhD Chief R&D Officer, Replimune 2002 in UK promise in oncolytic therapy GNCSF Phase III melanoma 2015 M&A with Amgen oncolytic therapy remains non effecting on immune response data is key for commercialization 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Ann Silk, MD Physician, Dana Farber-Brigham and Women’s Cancer Center, HMS Which person gets oncolytics virus if patient has immune supression due to other indications Safety of oncolytic virus greater than Systemic treatment

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

@pharma_BI

@AVIVA1950

amazing Conference on the frontier od Science Cell & Gene Therapy

@MGB

top programs for ALS, Brain genetic vasculopathologies and Occular, MEE

@pharma_BI

@AVIVA1950

Quote Tweet

Pearl Freier

@PearlF

 · 21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021



Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Merit Cudkowicz, MD Chief of Neurology, MGH ALS – Man 1in 300, Women 1 in 400, next decade increase 7%  10% ALS is heredity 160 pharma in ALS space diagnosis is late 1/3 of people are not diagnosed active community for clinical trials @pharma_BI@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Adam Koppel, MD, PhD Managing Director, Bain Capital Life Sciences What acquirers are looking for?? What is the next generation vs what is real where is the industry going?

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Debby Baron, Worldwide Business Development, Pfizer  Scalability and manufacturing regulatory conversations, clinical programs safety in parallel to planning getting drug to patients

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Marianne De Backer, PhD Head of Strategy, BD & Licensing, Bayer Absolute Leadership: Gene editing, gene therapy, via acquisition and alliances Operating model of the acquired company discussed acquired continue independence

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Sean Nolan Board Chairman, Encoded Therapeutics & Affinia Executive Chairman Jaguar Gene Therapy Istari Oncology As acquiree multiple M&A acquirer looks at integration and cultures companies  Traditional integration vs acquisition 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Debby Baron, Worldwide Business Development, Pfizer  CGT is an important area Pfizer is active looking for innovators, advancing forward programs of innovation with the experience Pfizer has internally 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Marianne De Backer, PhD Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer Absolute Leadership in Gene editing, gene therapy, via acquisition and strategic alliance 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

22h

2 people unfollowed me // automatically checked by

fllwrs – keep track of who follows and unfollows you on twitter

fllwrs is the easiest way to keep track of your twitter followers

fllwrs.com

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Manny Simons, PhD CEO, Akouos Biology across species nerve ending in the cochlea engineer out of the caspid, lowest dose possible, get desired effect by vector use, 2022 new milestones

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Mathew Pletcher, PhD SVP, Head of Gene Therapy Research and Technical Operations, Astellas Continue to explore large animal guinea pig not the mice, not primates (ethical issues) for understanding immunogenicity and immune response 

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Mathew Pletcher, PhD SVP, Head of Gene Therapy Research and Technical Operations, Astellas Work with diseases poorly understood, collaborations needs example of existing: DMD is a great example explain dystrophin share placedo data 

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Rick Modi CEO, Affinia Therapeutics Speed R&D Speed better gene construct get to clinic with better design vs ASAP Data sharing clinical experience patients selection, vector selection, mitigation, patient type specific

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies big pharma therapeutics not one drug across Tx areas: cell, gene iodine therapy collective learning infrastructure development Acquisitions growth # applications for scaling 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Rick Modi CEO, Affinia Therapeutics Copy, paste EDIT from product A to B novel vectors variant of vector coder optimization choice of indication is critical exploration on larger populations Speed to R&D to better gene construct get

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Louise Rodino-Klapac, PhD EVP, Chief Scientific Officer, Sarepta Therapeutics AV based platform 15 years in development 1 disease indication vs more than one indication stereotype, analytics as hurdle 1st was 10 years 2nd was 3 years

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Katherine High, MD President, Therapeutics, AskBio Three drugs approved in Europe in the CGT Regulatory Infrastructure CGT drug approval – as new class of therapeutics Participants investigators, regulators, patients i.e., MDM 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA Immune modulators Immunotherapy Genome editing can make use of viral vectors future technologies nanoparticles and liposome encapsulation 50% more staff

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA Recover Work load for the pandemic Gene Therapies IND application remained flat Rare diseases urgency remains Guidance T-Cell therapy vs Regulation

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA June 2020 belief that vaccine challenge manufacture scaling up FDA did not predicted the efficacy of mRNA vaccine vs other approaches expected to work

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Jim Holland CEO, http://Backcountry.com Parkinson patient Constraints by regulatory on participation in clinical trial wish to take Information dissemination is critical 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Patricia Musolino, MD, PhD Co-Director Pediatric Stroke and Cerebrovascular Program What is the Power of One – the impact that a patient can have on their own destiny connecting with other participants in same trial can be beneficial

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Barbara Lavery Chief Program Officer, ACGT Foundation Patient has the knowledge of the symptoms and recording all input needed for diagnosis by multiple clinicians Early application for CGT

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Sarah Beth Thomas, RN Professional Development Manager, BWH Outcome is unknown, hope for good, support with resources all advocacy groups, 

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Jack Hogan Patient, MEE Constraints by regulatory on participation in #clinicaltrials advance stage is approved participation Patients to determine the level of #risk they wish to take 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Barbara Lavery Chief Program Officer, ACGT Foundation Advocacy agency beginning of work Global Genes educational content and out reach to access the information

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies Modality one time intervention, long duration of impart, reimbursement, ecosystem FDA works by indications and risks involved, Standards manufacturing payments over time payers

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies Promise of CGT realized, what part? #FDA role and interaction in CGT #Manufacturing aspects which is critical

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Julian Harris, MD Partner, Deerfield Hope that CGT emerging, how therapies work, #neuro, #muscular, #ocular, #genetic diseases of #liver and of #heart revolution for the industry 900 #IND application 25 approvals #Economic driver 

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Luk Vandenberghe, PhD Grousbeck Family Chair, Gene Therapy, MEE Associate Professor, Ophthalmology, HMS #Pharmacology #Gene-Drug, Interface academic centers and industry many CGT drugs emerged in Academic center

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Ravi Thadhani, MD CAO, Mass General Brigham Professor, Medicine and Faculty Dean, HMS Role of #academia special to spear head the #Polygenic #therapy – multiple #genes involved, #plug-play #delivery

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Nino Chiocca, MD, PhD Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH #Oncolytic #Viruses triple threats #Toxic, #braintumors #immunological requires #combination #therapies with #anticancer

@pharma_BI

@AVIVA1950

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 19, 2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

17h

Will point of care production become a reality? “Short answer is yes” says Rupa Pike PhD, Director, Enterprise Science & Innovation Partnerships,

@thermofisher

. #WMIF2021 #GCTManufacturing

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Novartis Gene Therapies

@NovartisGene

·

May 18

The field of #genetherapy is growing. New therapies will come to market for rare and chronic diseases, and new therapies will drive scientific innovation and economic growth. #WMIF2021 (2/6)

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Aviva Lev-Ari

@AVIVA1950

·

15h

Very creative two targets

@ScaddenLab

@pharma_BI

@AVIVA1950

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 16h

A behind the scenes look at David Scadden, MD @ScaddenLab presenting his FIRST LOOK: Regenerating T Cell Immunity #WMIF2021 #GCT #Tcells

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Mass General Brigham Innovation

@MGBInnovation

·

16h

In our First Look sessions clinicians/researchers from Harvard-affiliated hospitals highlight the potential of their research & new technologies. Next we’ll hear from Khalid Shah PhD, Vice Chair of Research

@BWHNeurosurgery

#WMIF2021 https://bwhclinicalandresearchnews.org/2021/05/11/look-whos-talking-world-medical-innovation-forum-first-look-speakers/…

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

19h

“Entrepreneurial Growth | Oncolytic Virus” panel, moderated by Reid Huber PhD, Partner

@ThirdRockV

, discusses how small companies can address the challenges of developing #oncolyticvirus therapies. #WMIF2021

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Novartis Gene Therapies

@NovartisGene

·

May 19

The World Medical Innovation Forum is here! During his fireside chat, our President Dave Lennon shares the immense promise ahead for #genetherapy.

@MGBInnovation

#WMIF2021

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Mass General Brigham Innovation

@MGBInnovation

·

May 18

Tomorrow is Day 1 of #WMIF2021! Hear from the world-renowned CEOs, investors, clinicians and scientists bringing game-changing discoveries and insights to #GCT. Register to attend today: https://worldmedicalinnovation.org/register/

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Novartis Gene Therapies

@NovartisGene

·

May 18

We’re at

@MGBInnovation

‘s World Medical Innovation Forum this week, discussing the future of #genetherapy. Here are our five predictions for where the industry is headed. #WMIF2021 (1/6)

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Mass General Brigham Innovation

@MGBInnovation

·

23h

Some incredible #visualnotes from this morning’s co-chair’s panel “The Grand Challenge of Widespread GCT Patient Benefits” #WMIF2021 #GCT #geneandcelltherapy

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Mass General Brigham Innovation

@MGBInnovation

·

May 17

The World Medical Innovation Forum #WMIF2021 is just two days away! Join us to hear the latest in #geneandcelltherapy #healthcare innovation. https://worldmedicalinnovation.org/register/

You Retweeted

BrighamResearch

@BrighamResearch

·

May 16

“We anticipate that our engineered tumor cell platform will have major contributions in finding a cure for #glioblastoma patients,” says

@khalidshahs

 of

@BWHNeurosurgery

. Catch a preview of his #WMIF2021 First Look talk here: https://fal.cn/3fpUL

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Mass Eye and Ear

@MassEyeAndEar

·

22h

Dr. Eric Pierce

@MassEyeAndEar

@HMSeye

explains at #WMIF2021 why the first FDA-approved gene therapy for inherited disease was for an inherited retinal degeneration, and what lessons have been learned from the success of that treatment.

Mass General Brigham Innovation

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Mass General Brigham Innovation

@MGBInnovation

·

22h

Ravi Thadhani, CAO @MassGeneralBrigham and Juergen Eckhardt, Head of

@LeapsbyBayer

, will be announcing the 2021 Innovation Discovery Grants at #WMIF2021 tomorrow, 5/20 @ 2:00 pm Eastern. https://worldmedicalinnovation.org

Quote Tweet

Leaps by Bayer

@LeapsByBayer

 · 22h

Together with @BayerPharma, we are pleased to be part of #WMIF2021, organized by @MassGenBrigham. This year’s event focuses on the transformative potential of #cellandgene therapy (#GCT).

Show this thread

 

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Mass General Brigham Innovation

@MGBInnovation

·

20h

Welcome back! Our next #WMIF2021 panel, Oncolytic Viruses in #Cancer | Curing #Melanoma and Beyond, features panelists from

@BrighamWomens

@Replimune

@EikonTX

@Amgen

and

@DanaFarber

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Novartis Gene Therapies

@NovartisGene

·

22h

“We are more committed to our mission than ever before – laser-focused on realizing the transformative potential of #genetherapy for patients.” – Dave Lennon, President, during #WMIF2021

Outstanding researcher and speaker

@pharma_BI

@AVIVA1950

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 21h

Patricia Musolino, MD PhD, Co-Director Pediatric Stroke and Cerebrovascular Program at MGH, discusses her work developing #genetherapy treatments for cerebral genetic vasculopathies #GCT #geneandcelltherapy #WMIF2021

1

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Mass Eye and Ear

@MassEyeAndEar

·

23h

Happening now at #WMIF2021.

@MassEyeAndEar

chief and

@HMSeye

chair Dr. Joan Miller moderates a panel on AAV gene therapy featuring director of Inherited Retinal Disorders Service and Ocular Genomics Institute, Dr. Eric Pierce.

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 23h

Our “AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy” panelists have taken the stage. #WMIF2021 @MassEyeAndEar @REGENXBIO @spark_tx @NovartisGene

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CRISPR Therapeutics

@CRISPRTX

·

19h

Attending

@MGBInnovation

World Medical Innovation Forum? Tune in to hear our CEO

@CRISPRSam

speak tomorrow at 3:25pm ET on innovations in cell and gene therapy, followed by a Q&A. Learn more: https://bit.ly/3eWb66R #WMIF2021

4

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Biogen

@biogen

·

15h

We are proud sponsors of the Virtual World Medical Innovation Forum (#WMIF2021). This year’s program will focus on the impact of gene and cell therapy as a way to potentially advance quality patient care, reduce cost and improve outcomes. Learn more:

World Medical Innovation Forum

worldmedicalinnovation.org

You Retweeted

Pearl Freier

@PearlF

·

16h

Jonathan Kraft introducing #wmif2021 session with Pfizer CSO & president of R&D Mikael Dolsten and MGH oncologist & chair of MGH Cancer Center Daniel Haber.

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Aviva Lev-Ari

@AVIVA1950

·

15h

MEE is the leader in cell therapy for retina genetic disease

Quote Tweet

Tracy Doyle

@doylet

 · May 19

Great discussion to open #WMIF2021 on the patient impact of #GCT @MGBInnovation World Medical Innovation Forum twitter.com/AVIVA1950/stat…

You Retweeted

Pearl Freier

@PearlF

·

May 19

Tuning into

@MGBInnovation

#WMIF2021 cell & gene therapy meeting.

@NovartisGene

president Dave Lennon & Deerfield partner Julian Harris having a “fireside chat.” Dave/Novartis: sees gene therapy as driver for economy generating need for highly skilled workers Incl manufacturing

2

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Pearl Freier

@PearlF

·

17h

Kite Pharma CEO (Gilead subsidiary) Christi Shaw said there are 120 biopharma companies working on CAR-T cell therapy & they are continuing to look for new partnerships. She also mentioned logistical challenges currently getting to Israel & helping patients there. #WMIF2021

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Pearl Freier

@PearlF

·

15h

Dolsten/Pfizer discussing their partnership with Ionis.https://ir.ionispharma.com/news-releases/news-release-details/ionis-and-akcea-announce-pfizer-has-initiated-phase-2b-clinical… #wmif2021

Ionis and Akcea announce that Pfizer has initiated a Phase 2b clinical study of vupanorsen (AKCEA…

The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.’s business for stockholders, potential investors, and financial analysts.

ir.ionispharma.com

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Pearl Freier

@PearlF

·

23h

FDA’s Dir of Center for Biologics Evaluation & Research Peter Marks interviewed by Vicki Sato- chairwoman of Vir Biotechnology, ex Vertex president & ex Biogen VP Research. Around June ’20, started 2c progress in covid vaccines w/ enough candidates moving forward #WMIF2021 1/n

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Tracy Doyle

@doylet

·

23h

FDA staffing up on gene therapies personnel by 50% says Peter Marks, MD, PhD, Center for Biologics Evaluation and Research

@US_FDA

at #WMIF2021

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Mass General Brigham Innovation

@MGBInnovation

·

18h

“Once you work on cell and gene therapy, its really hard to go back and work on anything else” says moderator Marcela Maus, MD PhD in our “CAR-T | Lessons Learned | What’s Next” panel #WMIF2021 #GCT #geneandcelltherapy

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Pearl Freier

@PearlF

·

20h

Ex Merck president R&D Roger Perlmutter is now Eikon Therapeutics CEO & is on #WMIF2021 oncolytic virus in cancer panel w/Amgen EVP R&D David Reese, ex BioVex CTO (T-VEC inventor

@robertcoffin3

now

@Replimune

founder/president, Dana-Farber physician Ann Silk, BWH’s Nino Chiocca

You Retweeted

Novartis Gene Therapies

@NovartisGene

·

May 18

During this week’s World Medical Innovation Forum with

@MassGenBrigham

, join our leaders for panels and presentations discussing what’s next for #genetherapy and the key trends shaping the industry as it evolves. #WMIF2021 https://bit.ly/3eYYls4

59 views

0:24 / 0:36

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Pearl Freier

@PearlF

·

16h

Dolsten/Pfizer discussed covid vaccines and real world evidence study in Israel. Was sole provider of vaccines in Israel. 95%-98% efficacy replicated in real world. Well above 90% efficacy in asymptomatic disease. #wmif2021

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Tracy Doyle

@doylet

·

18h

Is CART-T therapy still an industry priority? Panelists say yes! Join us to hear more at the

@MGBInnovation

#WMIF2021

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Pearl Freier

@PearlF

·

18h

CAR-T #WMIF2021 panel w/ MGH’s

@MarcelaMaus

,

@Atarabio

EVP R&D

@jdupontmd

, BMS SVP Hematology/Oncology & Cell Therapy

@KristenHege

,

@KitePharma

CEO Christi Shaw, Novartis Global Head Cell & Gene

@Stefanhendriks5

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Mass General Brigham Innovation

@MGBInnovation

·

16h

ICYMI: An illustration depicting the “AAV Delivery” panel discussion about advances in the area of #AAVGeneTherapy delivery. Thank you to the panelists from

@MGHNeurology

,

@CureFA_org

,

@AstellasUS

and

@AkouosInc

. #geneandcelltherapy #GCT #WMIF2021



Aviva Lev-Ari

@AVIVA1950

·

16h

Like that presentation a lot

@pharma_BI

@AVIVA1950

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 22h

Casey Maguire PhD, Associate Professor of Neurology, at the podium to present his work developing improved #genetherapy vectors. #WMIF2021 “First Look: Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification”

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

22h

Casey Maguire PhD, Associate Professor of Neurology, at the podium to present his work developing improved #genetherapy vectors. #WMIF2021 “First Look: Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification”

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Aviva Lev-Ari

@AVIVA1950

·

16h

Best interview of a CSO in the history of Big Pharma

@Pharma_BI

@AVIVA1950

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 16h

Mikael Dolsten, MD PhD, CSO & President, Worldwide Research, Development and Medical @pfizer takes the stage for a Fireside Chat, moderated by @MGHCancerCenter Daniel Haber, MD, PhD. “Pfizer’s Future in Cell and Gene Therapy” #WMIF2021

You Retweeted

Pearl Freier

@PearlF

·

May 19

Dave Lennon/Novartis: manufacturing has been a roadblock for many cell & gene therapy companies. Expects to see more investments earlier. Engineering advances will unlock scale & address bigger & bigger patient populations. Oppty to ID patients early #WMIF2021

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Mass General Brigham Innovation

@MGBInnovation

·

19h

Nino Chiocca, MD PhD,

@BWHNeurosurgery

presents FIRST LOOK: Oncolytic Viruses: Turning Pathogens into Anticancer Agents #WMIF2021

You Retweeted

Pearl Freier

@PearlF

·

22h

M&A cell & gene therapy #WMIF2021 panel incl Bain Capital’s Adam Koppel, Bayer’s Head Strategy Business Development & Licensing

@MDDBacker

, Pfizer’s SVP Worldiwde BD Debbie Baron, Eli Lilly VP BD Ken Custer, ex AveXis CEO Sean Nolan now Affinia & Encoded Therapeutics Board Chair

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Pearl Freier

@PearlF

·

21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021

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Resilience

@IncResilience

·

17h

Happening now: our CEO, Rahul Singhvi, speaking at the virtual 2021 World Medical Innovation Forum: http://worldmedicalinnovation.org #WMIF2021 https://pic.twitter.com/Nyc2lXbvUR

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Pearl Freier

@PearlF

·

21h

Ken Custer/Eli Lilly-said they’re relatively new in cell & gene therapy. They invested in 1 of Sean Nolan’s (ex AveXis CEO) new companies,Jaguar Gene Therapy. Lilly’s legacy in neuroscience is noted & bought Prevail last yr. Clinical trial w/ Parkinson’s w/GBA1 mutation #wmif2021

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Mass General Brigham Innovation

@MGBInnovation

·

May 19

Jack Hogan, a patient

@MassEyeAndEar

, was the first in the U.S. to be approved for FDA gene therapy surgery. In 2018 he underwent therapy to treat retinitis pigmentosa by having a synthetic gene inserted into his retina. With improved eyesight he can now play sports #WMIF2021

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Tracy Doyle

@doylet

·

21h

The acquisition market in #GCT: looking for breakthroughs for patients, technologies for intractable diseases, manufacturing expertise, pioneering companies with deep experience — all for “the modality of the future”. M&A panel at #WMIF2021

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Pearl Freier

@PearlF

·

18h

Christi Shaw/Kite Pharma: Only 4 out of 10 patients eligible for CAR-T are being referred for CAR-T cell therapy by oncologists. The other 6 out of 10, referred to palliative care only. Consistency of manufacturing is also very important. #wmif2021 1/n

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Mass Eye and Ear

@MassEyeAndEar

·

22h

AAV gene therapy expert

@VandenbergheLuk

@HMSeye

@MassEyeAndEar

presents on the future potential of this revolutionary technology at #WMIF2021

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

@pharma_BI

@AVIVA1950

amazing Conference on the frontier od Science Cell & Gene Therapy

@MGB

top programs for ALS, Brain genetic vasculopathologies and Occular, MEE

@pharma_BI

@AVIVA1950

Quote Tweet

Pearl Freier

@PearlF

 · 21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021

Read Full Post »

Reporter: Danielle Smolyar, Research Assistant 3 – Text Analysis for 2.0 LPBI Group’s TNS #1 – 2020/2021 Academic Internship in Medical Test Analysis (MTA) 

Reporting on a Study published on July 6, 2021 by  Oregon Health & Science University

Recently, researchers have found many ways to manipulate and alter gene activity in specific cells. As a result of seeing this alteration, it has caused much development and progress in understanding cancer, brain function, and immunity.

IMAGE SOURCE: 3D-model of DNA. Credit: Michael Ströck/Wikimedia/ GNU Free Documentation Lic

Tissues and Organs are composed of cells that look the same but have different roles. For example, single-cell analysis allows us to research and test the cells within an organ or cancerous tumor. However, the single-cell study has its boundaries and limits in trying a more significant number of cells. This result is not an accurate data and analysis of the cells.

Andrew Adey, Ph.D., the senior author of a paper in Nature Biotechnology, https://www.nature.com/articles/s41587-021-00962-z

Mulqueen, R. M., Pokholok, D., O’Connell, B. L., Thornton, C. A., Zhang, F., O’Roak, B. J., Link, J., Yardımcı, G. G., Sears, R. C., Steemers, F. J., & Adey, A. C. (2021, July 5). High-content single-cell combinatorial indexing. Nature News. https://www.nature.com/articles/s41587-021-00962-z

states that the new method gives us the ability to have a ten-fold improvement in the amount of DNA produced from a single DNA sequence. A DNA sequence is composed of units which are called bases. The sequence puts the bases in chronological order for it to code correctly. 

To understand cancer better, single-cell studies are a crucial factor in doing so. Different cells catch on to other mutations in the DNA sequence in a cancerous tumor, which ultimately alters the DNA sequence. This results in tumor cells with new alterations, which could eventually spread to the rest of the body. 

Adey and his team provided evidence that the method they had created can show DNA alterations that have come from cells present in tumor samples from patients with pancreatic cancer. Adey stated,

quote “For example, you can potentially identify rare cell subtypes within a tumor that are resistant to therapy.” 

Abey and his team have been working with OHSU Knight Cancer Institute, and with them, they are testing a single-cell method to see if patients’ tumors have changed by doing chemo or drug therapy. 

This new method allows itself to create DNA libraries and fragments of DNA that helps analyze the different genes and mutations within the sequence. This method uses something called an enzymatic reaction that attaches primers to the end of each DNA fragment.  For the cells to be analyzed, each primer must be present on both ends of the fragment. 

As a result of this new method, all library fragments present must-have primers on both ends of the fragments. At the same time, it improves efficiency by reducing its sequencing  price overall, that these adapters can be used instead of the regular custom workflows. 

SOURCE

Original article:

Mulqueen, R.M., Pokholok, D., O’Connell, B.L. et al. High-content single-cell combinatorial indexing. Nat Biotechnol (2021). https://doi.org/10.1038/s41587-021-00962-z

Research categories – Cell biology, cancer-general, research, DNA Fragment TAGS- DNA, sequencing, cell fragments, single-cell

Other related articles published on this Open Access Online Scientific Journal include the following: 

Series B: Frontiers in Genomics Research

Series Content Consultant:

Larry H. Bernstein, MD, FCAP, Emeritus CSO, LPBI Group

Volume Content Consultant:

Prof. Marcus W. Feldman

BURNET C. AND MILDRED FINLEY WOHLFORD PROFESSOR IN THE SCHOOL OF HUMANITIES AND SCIENCES

Stanford University, Co-Director, Center for Computational, Evolutionary and Human Genetics (2012 – Present)

Latest in Genomics Methodologies for Therapeutics:

Gene Editing, NGS & BioInformatics,

Simulations and the Genome Ontology

2019

Volume Two

https://www.amazon.com/dp/B08385KF87

 

Part 4: Single Cell Genomics

Introduction to Part 4: Single Cell Genomics – Voice of Aviva Lev-Ari & Stephen Williams


4.1 The Science

4.1.1   Single-cell biology

Special | 05 July 2017

https://www.nature.com/collections/gbljnzchgg

4.1.2   The race to map the human body — one cell at a time, A host of detailed cell atlases could revolutionize understanding of cancer and other diseases

https://www.nature.com/news/the-race-to-map-the-human-body-one-cell-at-a-time-1.21508

4.1.3   Single-cell Genomics: Directions in Computational and Systems Biology – Contributions of Prof. Aviv Regev @Broad Institute of MIT and Harvard, Cochair, the Human Cell Atlas Organizing Committee with Sarah Teichmann of the Wellcome Trust Sanger Institute

Curator: Aviva Lev-Ari, PhD, RN

4.1.4   Cellular Genetics

https://www.sanger.ac.uk/science/programmes/cellular-genetics

4.1.5   Cellular Genomics

https://www.garvan.org.au/research/cellular-genomics

4.1.6   SINGLE CELL GENOMICS 2019 – sometimes the sum of the parts is greater than the whole, September 24-26, 2019, Djurönäset, Stockholm, Sweden http://www.weizmann.ac.il/conferences/SCG2019/single-cell-genomics-2019

Reporter: Aviva Lev-Ari, PhD, RN

4.1.7   Norwich Single-Cell Symposium 2019, Earlham Institute, single-cell genomics technologies and their application in microbial, plant, animal and human health and disease, October 16-17, 2019, 10AM-5PM

Reporter: Aviva Lev-Ari, PhD, RN

4.1.8   Newly Found Functions of B Cell

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

4.1.9 RESEARCH HIGHLIGHTS: HUMAN CELL ATLAS

https://www.broadinstitute.org/research-highlights-human-cell-atlas

4.2 Technologies and Methodologies

4.2.1   How to build a human cell atlas – Aviv Regev is a maven of hard-core biological analyses. Now she is part of an effort to map every cell in the human body.

Anna Nowogrodzki, 05 July 2017, Article tools

https://www.nature.com/news/how-to-build-a-human-cell-atlas-1.22239

4.2.2   Featuring Computational and Systems Biology Program at Memorial Sloan Kettering Cancer Center, Sloan Kettering Institute (SKI), The Dana Pe’er Lab

Reporter: Aviva Lev-Ari, PhD, RN

4.2.3   Genomic Diagnostics: Three Techniques to Perform Single Cell Gene Expression and Genome Sequencing Single Molecule DNA Sequencing

Curator: Aviva Lev-Ari, PhD, RN

4.2.4   Three Technology Leaders in Single Cell Sequencing: 10X Genomics, Illumina and MissionBio

Reporter: Aviva Lev-Ari, PhD, RN

4.2.5   scPopCorn: A New Computational Method for Subpopulation Detection and their Comparative Analysis Across Single-Cell Experiments

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

4.2.6   Nano-guided cell networks: new methods to detect intracellular signaling and implications

Curator: Stephen J. Williams, PhD

4.3 Clinical Aspects

4.3.1 Using single cell sequencing data to model the evolutionary history of a tumor.

Kim KI, Simon R.

BMC Bioinformatics. 2014 Jan 24;15:27. doi: 10.1186/1471-2105-15-27.

PMID:

4.3.2   eProceedings 2019 Koch Institute Symposium – 18th Annual Cancer Research Symposium – Machine Learning and Cancer, June 14, 2019, 8:00 AM-5:00 PM ET MIT Kresge Auditorium, 48 Massachusetts Ave, Cambridge, MA

Real Time Press Coverage: Aviva Lev-Ari, PhD, RN

4.3.3   The Impact of Heterogeneity on Single-Cell Sequencing

Samantha L. Goldman1,2, Matthew MacKay1,2, Ebrahim Afshinnekoo1,2,3, Ari M. Melnick4, Shuxiu Wu5,6 and Christopher E. Mason1,2,3,7*

https://www.frontiersin.org/articles/10.3389/fgene.2019.00008/full

4.3.4   Single-cell approaches to immune profiling

https://www.nature.com/articles/d41586-018-05214-w

4.3.5   Single-cell sequencing made simple. Data from thousands of single cells can be tricky to analyse, but software advances are making it easier.

by Jeffrey M. Perkel

https://www.nature.com/news/single-cell-sequencing-made-simple-1.22233

4.3.6  Single-cell RNA-seq helps in finding intra-tumoral heterogeneity in pancreatic cancer

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

4.3.7 Cancer Genomics: Multiomic Analysis of Single Cells and Tumor Heterogeneity

Curator: Stephen J. Williams, PhD

4.4 Business and Legal

4.4.1   iBioChips integrate diagnostic assays and cellular engineering into miniaturized chips that achieve cutting-edge sensitivity and high-throughput. We have resolved traditional biotech challenges with innovative biochip approaches

https://ibiochips.com/?gclid=Cj0KCQjwuLPnBRDjARIsACDzGL0wb6u79VHHkftodfApMYs-oxI-5cOZIBUaELdmd2wDOIk3W0OQg2caAqMyEALw_wcB

4.4.2   Targeted Single-Cell Solutions for High Impact Applications – Mission Bio’s Tapestri® Platform is the only technology that provides single-cell targeted DNA sequencing at single-base resolution.

Part 4: Summary – Single Cell Genomics – Voice of Stephen Williams

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