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Eight Subcellular Pathologies driving Chronic Metabolic Diseases – Methods for Mapping Bioelectronic Adjustable Measurements as potential new Therapeutics: Impact on Pharmaceuticals in Use

Eight Subcellular Pathologies driving Chronic Metabolic Diseases – Methods for Mapping Bioelectronic Adjustable Measurements as potential new Therapeutics: Impact on Pharmaceuticals in Use

Curators:

 

THE VOICE of Aviva Lev-Ari, PhD, RN

In this curation we wish to present two breaking through goals:

Goal 1:

Exposition of a new direction of research leading to a more comprehensive understanding of Metabolic Dysfunctional Diseases that are implicated in effecting the emergence of the two leading causes of human mortality in the World in 2023: (a) Cardiovascular Diseases, and (b) Cancer

Goal 2:

Development of Methods for Mapping Bioelectronic Adjustable Measurements as potential new Therapeutics for these eight subcellular causes of chronic metabolic diseases. It is anticipated that it will have a potential impact on the future of Pharmaceuticals to be used, a change from the present time current treatment protocols for Metabolic Dysfunctional Diseases.

According to Dr. Robert Lustig, M.D, an American pediatric endocrinologist. He is Professor emeritus of Pediatrics in the Division of Endocrinology at the University of California, San Francisco, where he specialized in neuroendocrinology and childhood obesity, there are eight subcellular pathologies that drive chronic metabolic diseases.

These eight subcellular pathologies can’t be measured at present time.

In this curation we will attempt to explore methods of measurement for each of these eight pathologies by harnessing the promise of the emerging field known as Bioelectronics.

Unmeasurable eight subcellular pathologies that drive chronic metabolic diseases

  1. Glycation
  2. Oxidative Stress
  3. Mitochondrial dysfunction [beta-oxidation Ac CoA malonyl fatty acid]
  4. Insulin resistance/sensitive [more important than BMI], known as a driver to cancer development
  5. Membrane instability
  6. Inflammation in the gut [mucin layer and tight junctions]
  7. Epigenetics/Methylation
  8. Autophagy [AMPKbeta1 improvement in health span]

Diseases that are not Diseases: no drugs for them, only diet modification will help

Image source

Robert Lustig, M.D. on the Subcellular Processes That Belie Chronic Disease

https://www.youtube.com/watch?v=Ee_uoxuQo0I

 

Exercise will not undo Unhealthy Diet

Image source

Robert Lustig, M.D. on the Subcellular Processes That Belie Chronic Disease

https://www.youtube.com/watch?v=Ee_uoxuQo0I

 

These eight Subcellular Pathologies driving Chronic Metabolic Diseases are becoming our focus for exploration of the promise of Bioelectronics for two pursuits:

  1. Will Bioelectronics be deemed helpful in measurement of each of the eight pathological processes that underlie and that drive the chronic metabolic syndrome(s) and disease(s)?
  2. IF we will be able to suggest new measurements to currently unmeasurable health harming processes THEN we will attempt to conceptualize new therapeutic targets and new modalities for therapeutics delivery – WE ARE HOPEFUL

In the Bioelecronics domain we are inspired by the work of the following three research sources:

  1. Biological and Biomedical Electrical Engineering (B2E2) at Cornell University, School of Engineering https://www.engineering.cornell.edu/bio-electrical-engineering-0
  2. Bioelectronics Group at MIT https://bioelectronics.mit.edu/
  3. The work of Michael Levin @Tufts, The Levin Lab
Michael Levin is an American developmental and synthetic biologist at Tufts University, where he is the Vannevar Bush Distinguished Professor. Levin is a director of the Allen Discovery Center at Tufts University and Tufts Center for Regenerative and Developmental Biology. Wikipedia
Born: 1969 (age 54 years), Moscow, Russia
Education: Harvard University (1992–1996), Tufts University (1988–1992)
Affiliation: University of Cape Town
Research interests: Allergy, Immunology, Cross Cultural Communication
Awards: Cozzarelli prize (2020)
Doctoral advisor: Clifford Tabin
Most recent 20 Publications by Michael Levin, PhD
SOURCE
SCHOLARLY ARTICLE
The nonlinearity of regulation in biological networks
1 Dec 2023npj Systems Biology and Applications9(1)
Co-authorsManicka S, Johnson K, Levin M
SCHOLARLY ARTICLE
Toward an ethics of autopoietic technology: Stress, care, and intelligence
1 Sep 2023BioSystems231
Co-authorsWitkowski O, Doctor T, Solomonova E
SCHOLARLY ARTICLE
Closing the Loop on Morphogenesis: A Mathematical Model of Morphogenesis by Closed-Loop Reaction-Diffusion
14 Aug 2023Frontiers in Cell and Developmental Biology11:1087650
Co-authorsGrodstein J, McMillen P, Levin M
SCHOLARLY ARTICLE
30 Jul 2023Biochim Biophys Acta Gen Subj1867(10):130440
Co-authorsCervera J, Levin M, Mafe S
SCHOLARLY ARTICLE
Regulative development as a model for origin of life and artificial life studies
1 Jul 2023BioSystems229
Co-authorsFields C, Levin M
SCHOLARLY ARTICLE
The Yin and Yang of Breast Cancer: Ion Channels as Determinants of Left–Right Functional Differences
1 Jul 2023International Journal of Molecular Sciences24(13)
Co-authorsMasuelli S, Real S, McMillen P
SCHOLARLY ARTICLE
Bioelectricidad en agregados multicelulares de células no excitables- modelos biofísicos
Jun 2023Revista Española de Física32(2)
Co-authorsCervera J, Levin M, Mafé S
SCHOLARLY ARTICLE
Bioelectricity: A Multifaceted Discipline, and a Multifaceted Issue!
1 Jun 2023Bioelectricity5(2):75
Co-authorsDjamgoz MBA, Levin M
SCHOLARLY ARTICLE
Control Flow in Active Inference Systems – Part I: Classical and Quantum Formulations of Active Inference
1 Jun 2023IEEE Transactions on Molecular, Biological, and Multi-Scale Communications9(2):235-245
Co-authorsFields C, Fabrocini F, Friston K
SCHOLARLY ARTICLE
Control Flow in Active Inference Systems – Part II: Tensor Networks as General Models of Control Flow
1 Jun 2023IEEE Transactions on Molecular, Biological, and Multi-Scale Communications9(2):246-256
Co-authorsFields C, Fabrocini F, Friston K
SCHOLARLY ARTICLE
Darwin’s agential materials: evolutionary implications of multiscale competency in developmental biology
1 Jun 2023Cellular and Molecular Life Sciences80(6)
Co-authorsLevin M
SCHOLARLY ARTICLE
Morphoceuticals: Perspectives for discovery of drugs targeting anatomical control mechanisms in regenerative medicine, cancer and aging
1 Jun 2023Drug Discovery Today28(6)
Co-authorsPio-Lopez L, Levin M
SCHOLARLY ARTICLE
Cellular signaling pathways as plastic, proto-cognitive systems: Implications for biomedicine
12 May 2023Patterns4(5)
Co-authorsMathews J, Chang A, Devlin L
SCHOLARLY ARTICLE
Making and breaking symmetries in mind and life
14 Apr 2023Interface Focus13(3)
Co-authorsSafron A, Sakthivadivel DAR, Sheikhbahaee Z
SCHOLARLY ARTICLE
The scaling of goals from cellular to anatomical homeostasis: an evolutionary simulation, experiment and analysis
14 Apr 2023Interface Focus13(3)
Co-authorsPio-Lopez L, Bischof J, LaPalme JV
SCHOLARLY ARTICLE
The collective intelligence of evolution and development
Apr 2023Collective Intelligence2(2):263391372311683SAGE Publications
Co-authorsWatson R, Levin M
SCHOLARLY ARTICLE
Bioelectricity of non-excitable cells and multicellular pattern memories: Biophysical modeling
13 Mar 2023Physics Reports1004:1-31
Co-authorsCervera J, Levin M, Mafe S
SCHOLARLY ARTICLE
There’s Plenty of Room Right Here: Biological Systems as Evolved, Overloaded, Multi-Scale Machines
1 Mar 2023Biomimetics8(1)
Co-authorsBongard J, Levin M
SCHOLARLY ARTICLE
Transplantation of fragments from different planaria: A bioelectrical model for head regeneration
7 Feb 2023Journal of Theoretical Biology558
Co-authorsCervera J, Manzanares JA, Levin M
SCHOLARLY ARTICLE
Bioelectric networks: the cognitive glue enabling evolutionary scaling from physiology to mind
1 Jan 2023Animal Cognition
Co-authorsLevin M
SCHOLARLY ARTICLE
Biological Robots: Perspectives on an Emerging Interdisciplinary Field
1 Jan 2023Soft Robotics
Co-authorsBlackiston D, Kriegman S, Bongard J
SCHOLARLY ARTICLE
Cellular Competency during Development Alters Evolutionary Dynamics in an Artificial Embryogeny Model
1 Jan 2023Entropy25(1)
Co-authorsShreesha L, Levin M
5

5 total citations on Dimensions.

Article has an altmetric score of 16
SCHOLARLY ARTICLE
1 Jan 2023BIOLOGICAL JOURNAL OF THE LINNEAN SOCIETY138(1):141
Co-authorsClawson WP, Levin M
SCHOLARLY ARTICLE
Future medicine: from molecular pathways to the collective intelligence of the body
1 Jan 2023Trends in Molecular Medicine
Co-authorsLagasse E, Levin M

THE VOICE of Dr. Justin D. Pearlman, MD, PhD, FACC

PENDING

THE VOICE of  Stephen J. Williams, PhD

Ten TakeAway Points of Dr. Lustig’s talk on role of diet on the incidence of Type II Diabetes

 

  1. 25% of US children have fatty liver
  2. Type II diabetes can be manifested from fatty live with 151 million  people worldwide affected moving up to 568 million in 7 years
  3. A common myth is diabetes due to overweight condition driving the metabolic disease
  4. There is a trend of ‘lean’ diabetes or diabetes in lean people, therefore body mass index not a reliable biomarker for risk for diabetes
  5. Thirty percent of ‘obese’ people just have high subcutaneous fat.  the visceral fat is more problematic
  6. there are people who are ‘fat’ but insulin sensitive while have growth hormone receptor defects.  Points to other issues related to metabolic state other than insulin and potentially the insulin like growth factors
  7. At any BMI some patients are insulin sensitive while some resistant
  8. Visceral fat accumulation may be more due to chronic stress condition
  9. Fructose can decrease liver mitochondrial function
  10. A methionine and choline deficient diet can lead to rapid NASH development

 

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Bacterial multidrug resistance problem solved by a broad-spectrum synthetic antibiotic

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

There is an increasing demand for new antibiotics that effectively treat patients with refractory bacteremia, do not evoke bacterial resistance, and can be readily modified to address current and anticipated patient needs. Recently scientists described a promising compound of COE (conjugated oligo electrolytes) family, COE2-2hexyl, that exhibited broad-spectrum antibacterial activity. COE2-2hexyl effectively-treated mice infected with bacteria derived from sepsis patients with refractory bacteremia, including a CRE K. pneumoniae strain resistant to nearly all clinical antibiotics tested. Notably, this lead compound did not evoke drug resistance in several pathogens tested. COE2-2hexyl has specific effects on multiple membrane-associated functions (e.g., septation, motility, ATP synthesis, respiration, membrane permeability to small molecules) that may act together to abrogate bacterial cell viability and the evolution of drug-resistance. Impeding these bacterial properties may occur through alteration of vital protein–protein or protein-lipid membrane interfaces – a mechanism of action distinct from many membrane disrupting antimicrobials or detergents that destabilize membranes to induce bacterial cell lysis. The diversity and ease of COE design and chemical synthesis have the potential to establish a new standard for drug design and personalized antibiotic treatment.

Recent studies have shown that small molecules can preferentially target bacterial membranes due to significant differences in lipid composition, presence of a cell wall, and the absence of cholesterol. The inner membranes of Gram-negative bacteria are generally more negatively charged at their surface because they contain more anionic lipids such as cardiolipin and phosphatidylglycerol within their outer leaflet compared to mammalian membranes. In contrast, membranes of mammalian cells are largely composed of more-neutral phospholipids, sphingomyelins, as well as cholesterol, which affords membrane rigidity and ability to withstand mechanical stresses; and may stabilize the membrane against structural damage to membrane-disrupting agents such as COEs. Consistent with these studies, COE2-2hexyl was well tolerated in mice, suggesting that COEs are not intrinsically toxic in vivo, which is often a primary concern with membrane-targeting antibiotics. The COE refinement workflow potentially accelerates lead compound optimization by more rapid screening of novel compounds for the iterative directed-design process. It also reduces the time and cost of subsequent biophysical characterization, medicinal chemistry and bioassays, ultimately facilitating the discovery of novel compounds with improved pharmacological properties.

Additionally, COEs provide an approach to gain new insights into microbial physiology, including membrane structure/function and mechanism of drug action/resistance, while also generating a suite of tools that enable the modulation of bacterial and mammalian membranes for scientific or manufacturing uses. Notably, further COE safety and efficacy studies are required to be conducted on a larger scale to ensure adequate understanding of the clinical benefits and risks to assure clinical efficacy and toxicity before COEs can be added to the therapeutic armamentarium. Despite these limitations, the ease of molecular design, synthesis and modular nature of COEs offer many advantages over conventional antimicrobials, making synthesis simple, scalable and affordable. It enables the construction of a spectrum of compounds with the potential for development as a new versatile therapy for the emergence and rapid global spread of pathogens that are resistant to all, or nearly all, existing antimicrobial medicines.

References:

https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(23)00026-9/fulltext#%20

https://pubmed.ncbi.nlm.nih.gov/36801104/

https://www.sciencedaily.com/releases/2023/02/230216161214.htm

https://www.nature.com/articles/s41586-021-04045-6

https://www.nature.com/articles/d43747-020-00804-y

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Mimicking vaginal cells and microbiome interactions on chip microfluidic culture

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

Scientists at Harvard University’s Wyss Institute for Biologically Inspired Engineering have developed the world’s first “vagina-on-a-chip,” which uses living cells and bacteria to mimic the microbial environment of the human vagina. It could help to test drugs against bacterial vaginosis, a common microbial imbalance that makes millions of people more susceptible to sexually transmitted diseases and puts them at risk of preterm delivery when pregnant. Vaginal health is difficult to study in a laboratory setting partly because laboratory animals have “totally different microbiomes” than humans. To address this, scientists have created an unique chip, which is an inch-long, rectangular polymer case containing live human vaginal tissue from a donor and a flow of estrogen-carrying material to simulate vaginal mucus.

The organs-on-a-chip mimic real bodily function, making it easier to study diseases and test drugs. Previous examples include models of the lungs and the intestines. In this case, the tissue acts like that of a real vagina in some important ways. It even responds to changes in estrogen by adjusting the expression of certain genes. And it can grow a humanlike microbiome dominated by “good” or “bad” bacteria. The researchers have demonstrated that Lactobacilli growing on the chip’s tissue help to maintain a low pH by producing lactic acid. Conversely, if the researchers introduce Gardnerella, the chip develops a higher pH, cell damage and increased inflammation: classic bacterial vaginosis signs. So, the chip can demonstrate how a healthy / unhealthy microbiome affects the vagina.

The next step is personalization or subject specific culture from individuals. The chip is a real leap forward, it has the prospect of testing how typical antibiotic treatments against bacterial vaginosis affect the different bacterial strains. Critics of organ-on-a-chip technology often raise the point that it models organs in isolation from the rest of the body. There are limitations such as many researchers are interested in vaginal microbiome changes that occur during pregnancy because of the link between bacterial vaginosis and labor complications. Although the chip’s tissue responds to estrogen, but it does not fully mimic pregnancy without feedback loops from other organs. The researchers are already working on connecting the vagina chip to a cervix chip, which could better represent the larger reproductive system.

All these information indicate that the human vagina chip offers a new model to study host-vaginal microbiome interactions in both optimal and non-optimal states, as well as providing a human relevant preclinical model for development and testing of reproductive therapeutics, including live bio-therapeutics products for bacterial vaginosis. This microfluidic human vagina chip that enables flow through an open epithelial lumen also offers a unique advantage for studies on the effect of cervicovaginal mucus on vaginal health as clinical mucus samples or commercially available mucins can be flowed through this channel. The role of resident and circulating immune cells in host-microbiome interactions also can be explored by incorporating these cells into the vagina chip in the future, as this has been successfully done in various other organ chip models.

References:

https://www.scientificamerican.com/article/first-vagina-on-a-chip-will-help-researchers-test-drugs/

https://www.webmd.com/infertility-and-reproduction/news/20230209/scientists-create-vagina-on-chip-what-to-know

https://www.livescience.com/vagina-on-a-chip

https://link.springer.com/article/10.1186/s40168-022-01400-1

https://www.nature.com/articles/s41585-022-00717-8

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2022 FDA Drug Approval List, 2022 Biological Approvals and Approved Cellular and Gene Therapy Products

 

 

Reporter: Aviva Lev-Ari, PhD, RN

SOURCE

Tal Bahar’s post on LinkedIn on 1/17/2023

Novel Drug Approvals for 2022

FDA’s Center for Drug Evaluation and Research (CDER)

New Molecular Entities (“NMEs”)

  • Some of these products have never been used in clinical practice. Below is a listing of new molecular entities and new therapeutic biological products that CDER approved in 2022. This listing does not contain vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products that the Center for Biologics Evaluation and Research approved in 2022. 
  • Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace. See Drugs@FDA for information about all of CDER’s approved drugs and biological products. 

Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that FDA had not previously approved, either as a single ingredient drug or as part of a combination product. These products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that FDA has previously approved. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act. 

INNOVATION   PREDICTABILITY   ACCESS FDA’s Center for Drug Evaluation and Research

January 2023

Table of Contents

 SOURCE

2022 Biological Approvals

The Center for Biologics Evaluation and Research (CBER) regulates products under a variety of regulatory authorities.  See the Development & Approval Process page for a description of what products are approved as Biologics License Applications (BLAs), Premarket Approvals (PMAs), New Drug Applications (NDAs) or 510Ks.

Biologics License Applications and Supplements

New BLAs (except those for blood banking), and BLA supplements that are expected to significantly enhance the public health (e.g., for new/expanded indications, new routes of administration, new dosage formulations and improved safety).

Other Applications Approved or Cleared by the Center for Biologics Evaluation and Research (CBER)

Medical devices involved in the collection, processing, testing, manufacture and administration of licensed blood, blood components and cellular products.

Key Resources

SOURCE

https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2022-biological-approvals

 

Approved Cellular and Gene Therapy Products

Below is a list of licensed products from the Office of Tissues and Advanced Therapies (OTAT).


Approved Products


 

Resources For You


SOURCE

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

 

2022 forecast: Cell, gene therapy makers push past regulatory, payer hurdles to set up high hopes for next year

There are five FDA-approved CAR-T treatments for blood cancers and two gene therapies to treat rare diseases now on the market in the U.S. The late-stage pipeline could produce several more cancer CAR-Ts and gene therapies to treat a range of diseases.

RELATED: ASH: Bristol Myers’ Breyanzi, Gilead’s Yescarta lock horns in race to move CAR-T therapy to earlier lymphoma

One of the biggest races to watch in the cell therapy space will be that between Gilead Sciences’ Yescarta and Bristol Myers Squibb’s Breyanzi, both of which are gunning to move their CAR-Ts into earlier lines of treatment in large B-cell lymphoma (LBCL). At ASH, both companies rolled out impressive data from their trials in the second-line setting, but Gilead could have the upper hand by virtue of its three-year head start in the market, analysts said. Gilead expects to hear from the FDA on a label expansion in the second-line setting in April.

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Cancer Policy Related News from Washington DC and New NCI Appointments

Reportor: Stephen J. Williams, PhD.

Article ID #297: Cancer Policy Related News from Washington DC and New NCI Appointments. Published on 10/4/2022

WordCloud Image Produced by Adam Tubman

Biden to announce appointees to Cancer Panel, part of initiative to cut death rate

The president first launched the initiative in 2016 as vice president.

By Mary Kekatos

July 13, 2022, 3:00 PM

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America This Morning

America This Morning

President Joe Biden will announce Wednesday his appointees to the President’s Cancer Panel, ABC News can exclusively reveal.

The Cancer Panel is part of Biden’s Cancer Moonshot Initiative, which was relaunched in February, with a goal of slashing the national cancer death rate by 50% over the next 25 years.MORE: Biden relaunches cancer ‘moonshot’ initiative to help cut death rate

Biden will appoint Dr. Elizabeth Jaffee, Dr. Mitchel Berger and Dr. Carol Brown to the panel, which will advise him and the White House on how to use resources of the federal government to advance cancer research and reduce the burden of cancer in the United States.

Jaffee, who will serve as chair of the panel, is an expert in cancer immunology and pancreatic cancer, according to the White House. She is currently the deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University and previously led the American Association for Cancer Research.

PHOTO: In this Sept. 8, 2016, file photo, Dr. Elizabeth M. Jaffee of the Pancreatic Dream Team attends Stand Up To Cancer (SU2C), a program of the Entertainment Industry Foundation (EIF), in Hollywood, Calif.
In this Sept. 8, 2016, file photo, Dr. Elizabeth M. Jaffee of the Pancreatic Dream Team attends Stand Up To Cancer (SU2C), a program of the Entertainment Industry Foundation (EIF), in Hollywood, Calif.ABC Handout via Getty Images, FILE

Berger, a neurological surgeon, directs the University of California, San Francisco Brain Tumor Center and previously spent 23 years at the school as a professor of neurological surgery.

Brown, a gynecologic oncologist, is the senior vice president and chief health equity officer at Memorial Sloan Kettering Cancer Center in New York City. According to the White House, much of her career has been focused on eliminating cancer care disparities due to racial, ethnic, cultural or socioeconomic factors.

Additionally, First Lady Jill Biden, members of the Cabinet and other administration officials are holding a meeting Wednesday of the Cancer Cabinet, made up of officials across several governmental departments and agencies, the White House said.

The Cabinet will introduce new members and discuss priorities in the battle against cancer including closing the screening gap, addressing potential environmental exposures, reducing the number of preventable cancer and expanding access to cancer research.MORE: Long Island school district found to have higher rates of cancer cases: Study

It is the second meeting of the cabinet since Biden relaunched the initiative in February, which he originally began in 2016 when he was vice president.

Both Jaffee and Berger were members of the Blue Ribbon Panel for the Cancer Moonshot Initiative led by Biden.

The initiative has personal meaning for Biden, whose son, Beau, died of glioblastoma — one of the most aggressive forms of brain cancer — in 2015.

“I committed to this fight when I was vice president,” Biden said at the time, during an event at the White House announcing the relaunch. “It’s one of the reasons why, quite frankly, I ran for president. Let there be no doubt, now that I am president, this is a presidential, White House priority. Period.”

The initiative has several priority actions including diagnosing cancer sooner; preventing cancer; addressing inequities; and supporting patients, caregivers and survivors.

PHOTO: In this June 14, 2016, file photo, Dr. Carol Brown, physician at Memorial Sloan Kettering Cancer Center, gives a presentation, at The White House Summit on The United State of Women, in Washington, D.C.
In this June 14, 2016, file photo, Dr. Carol Brown, physician at Memorial Sloan Kettering Cancer Center, gives a presentation, at The White House Summit on The United State of Women, in Washington, D.C.NurPhoto via Getty Images, FILE

The White House has also issued a call to action to get cancer screenings back to pre-pandemic levels.

More than 9.5 million cancer screenings that would have taken place in 2020 were missed due to the COVID-19 pandemic, according to the National Institutes of Health.MORE: Louisiana’s ‘Cancer Alley’ residents in clean air fight

“We have to get cancer screenings back on track and make sure they’re accessible to all Americans,” Biden said at the time.

Since the first meeting of the Cancer Cabinet, the Centers for Disease Control and Prevention has issued more than $200 million in grants to cancer prevention programs, the Centers for Medicaid & Medicare Services implemented a new model to reduce the cost of cancer care, and the U.S. Patent and Trademark Office said it will fast-track applications for cancer immunotherapies.

ABC News’ Sasha Pezenik contributed to this report.

Biden to tap prominent Harvard cancer surgeon to head National Cancer Institute

Monica Bertagnolli brings leadership experience in cancer clinical trials funded by the $7 billion research agency

headshot of Monica Bertagnolli
Monica BertagnolliASCO; GLENN DAVENPORT

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President Joe Biden is expected to pick cancer surgeon Monica Bertagnolli as the next director of the National Cancer Institute (NCI). Bertagnolli, a physician-scientist at Brigham and Women’s Hospital, the Dana-Farber Cancer Center, and Harvard Medical School, specializes in gastrointestinal cancers and is well known for her expertise in clinical trials. She will replace Ned Sharpless, who stepped down as NCI director in April after nearly 5 years.

The White House has not yet announced the selection, first reported by STAT, but several cancer research organizations closely watching for the nomination have issued statements supporting Bertagnolli’s expected selection. She is “a national leader” in clinical cancer research and “a great person to take the job,” Sharpless told ScienceInsider.

With a budget of $7 billion, NCI is the largest component of the National Institutes of Health (NIH) and the world’s largest funder of cancer research. Its director is the only NIH institute director selected by the president. Bertagnolli’s expected appointment, which does not require Senate confirmation, drew applause from the cancer research community

Margaret Foti, CEO of the American Association for Cancer Research, praised Bertagnolli’s “appreciation for … basic research” and “commitment to ensuring that such treatment innovations reach patients … across the United States.” Ellen Sigal, chair and founder of Friends of Cancer Research, says Bertagnolli “brings expertise the agency needs at a true inflection point for cancer research.”

Bertagnolli, 63, will be the first woman to lead NCI. Her lab research on tumor immunology and the role of a gene called APC in colorectal cancer led to a landmark trial she headed showing that an anti-inflammatory drug can help prevent this cancer. In 2007, she became the chief of surgery at the Dana-Farber Brigham Cancer Center.

She served as president of the American Society of Clinical Oncology in 2018 and currently chairs the Alliance for Clinical Trials in Oncology, which is funded by NCI’s National Clinical Trials Network. The network is a “complicated” program, and “Monica will have a lot of good ideas on how to make it work better,” Sharpless says.

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One of Bertagnolli’s first tasks will be to shape NCI’s role in Biden’s reignited Cancer Moonshot, which aims to slash the U.S. cancer death rate in half within 25 years. NCI’s new leader also needs to sort out how the agency will mesh with a new NIH component that will fund high-risk, goal-driven research, the Advanced Research Projects Agency for Health (ARPA-H).

Bertagnolli will also head NCI efforts already underway to boost grant funding rates, diversify the cancer research workplace, and reduce higher death rates for Black people with cancer.

The White House recently nominated applied physicist Arati Prabhakar to fill another high-level science position, director of the White House Office of Science and Technology Policy (OSTP). But still vacant is the NIH director slot, which Francis Collins, acting science adviser to the president, left in December 2021. And the administration hasn’t yet selected the inaugural director of ARPA-H.

Correction, 22 July, 9 a.m.: This story has been updated to reflect that Francis Collins is acting science adviser to the president, not acting director of the White House Office of Science and Technology Policy.

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Reporter: Stephen J. Williams, Ph.D.

From: Heidi Rheim et al. GA4GH: International policies and standards for data sharing across genomic research and healthcare. (2021): Cell Genomics, Volume 1 Issue 2.

Source: DOI:https://doi.org/10.1016/j.xgen.2021.100029

Highlights

  • Siloing genomic data in institutions/jurisdictions limits learning and knowledge
  • GA4GH policy frameworks enable responsible genomic data sharing
  • GA4GH technical standards ensure interoperability, broad access, and global benefits
  • Data sharing across research and healthcare will extend the potential of genomics

Summary

The Global Alliance for Genomics and Health (GA4GH) aims to accelerate biomedical advances by enabling the responsible sharing of clinical and genomic data through both harmonized data aggregation and federated approaches. The decreasing cost of genomic sequencing (along with other genome-wide molecular assays) and increasing evidence of its clinical utility will soon drive the generation of sequence data from tens of millions of humans, with increasing levels of diversity. In this perspective, we present the GA4GH strategies for addressing the major challenges of this data revolution. We describe the GA4GH organization, which is fueled by the development efforts of eight Work Streams and informed by the needs of 24 Driver Projects and other key stakeholders. We present the GA4GH suite of secure, interoperable technical standards and policy frameworks and review the current status of standards, their relevance to key domains of research and clinical care, and future plans of GA4GH. Broad international participation in building, adopting, and deploying GA4GH standards and frameworks will catalyze an unprecedented effort in data sharing that will be critical to advancing genomic medicine and ensuring that all populations can access its benefits.

In order for genomic and personalized medicine to come to fruition it is imperative that data siloes around the world are broken down, allowing the international collaboration for the collection, storage, transferring, accessing and analying of molecular and health-related data.

We had talked on this site in numerous articles about the problems data siloes produce. By data siloes we are meaning that collection and storage of not only DATA but intellectual thought are being held behind physical, electronic, and intellectual walls and inacessible to other scientisits not belonging either to a particular institituion or even a collaborative network.

Scientific Curation Fostering Expert Networks and Open Innovation: Lessons from Clive Thompson and others

Standardization and harmonization of data is key to this effort to sharing electronic records. The EU has taken bold action in this matter. The following section is about the General Data Protection Regulation of the EU and can be found at the following link:

https://ec.europa.eu/info/law/law-topic/data-protection/data-protection-eu_en

Fundamental rights

The EU Charter of Fundamental Rights stipulates that EU citizens have the right to protection of their personal data.

Protection of personal data

Legislation

The data protection package adopted in May 2016 aims at making Europe fit for the digital age. More than 90% of Europeans say they want the same data protection rights across the EU and regardless of where their data is processed.

The General Data Protection Regulation (GDPR)

Regulation (EU) 2016/679 on the protection of natural persons with regard to the processing of personal data and on the free movement of such data. This text includes the corrigendum published in the OJEU of 23 May 2018.

The regulation is an essential step to strengthen individuals’ fundamental rights in the digital age and facilitate business by clarifying rules for companies and public bodies in the digital single market. A single law will also do away with the current fragmentation in different national systems and unnecessary administrative burdens.

The regulation entered into force on 24 May 2016 and applies since 25 May 2018. More information for companies and individuals.

Information about the incorporation of the General Data Protection Regulation (GDPR) into the EEA Agreement.

EU Member States notifications to the European Commission under the GDPR

The Data Protection Law Enforcement Directive

Directive (EU) 2016/680 on the protection of natural persons regarding processing of personal data connected with criminal offences or the execution of criminal penalties, and on the free movement of such data.

The directive protects citizens’ fundamental right to data protection whenever personal data is used by criminal law enforcement authorities for law enforcement purposes. It will in particular ensure that the personal data of victims, witnesses, and suspects of crime are duly protected and will facilitate cross-border cooperation in the fight against crime and terrorism.

The directive entered into force on 5 May 2016 and EU countries had to transpose it into their national law by 6 May 2018.

The following paper by the organiztion The Global Alliance for Genomics and Health discusses these types of collaborative efforts to break down data silos in personalized medicine. This organization has over 2000 subscribers in over 90 countries encompassing over 60 organizations.

Enabling responsible genomic data sharing for the benefit of human health

The Global Alliance for Genomics and Health (GA4GH) is a policy-framing and technical standards-setting organization, seeking to enable responsible genomic data sharing within a human rights framework.

he Global Alliance for Genomics and Health (GA4GH) is an international, nonprofit alliance formed in 2013 to accelerate the potential of research and medicine to advance human health. Bringing together 600+ leading organizations working in healthcare, research, patient advocacy, life science, and information technology, the GA4GH community is working together to create frameworks and standards to enable the responsible, voluntary, and secure sharing of genomic and health-related data. All of our work builds upon the Framework for Responsible Sharing of Genomic and Health-Related Data.

GA4GH Connect is a five-year strategic plan that aims to drive uptake of standards and frameworks for genomic data sharing within the research and healthcare communities in order to enable responsible sharing of clinical-grade genomic data by 2022. GA4GH Connect links our Work Streams with Driver Projects—real-world genomic data initiatives that help guide our development efforts and pilot our tools.

From the article on Cell Genomics GA4GH: International policies and standards for data sharing across genomic research and healthcare

Source: Open Access DOI:https://doi.org/10.1016/j.xgen.2021.100029PlumX Metrics

The Global Alliance for Genomics and Health (GA4GH) is a worldwide alliance of genomics researchers, data scientists, healthcare practitioners, and other stakeholders. We are collaborating to establish policy frameworks and technical standards for responsible, international sharing of genomic and other molecular data as well as related health data. Founded in 2013,3 the GA4GH community now consists of more than 1,000 individuals across more than 90 countries working together to enable broad sharing that transcends the boundaries of any single institution or country (see https://www.ga4gh.org).In this perspective, we present the strategic goals of GA4GH and detail current strategies and operational approaches to enable responsible sharing of clinical and genomic data, through both harmonized data aggregation and federated approaches, to advance genomic medicine and research. We describe technical and policy development activities of the eight GA4GH Work Streams and implementation activities across 24 real-world genomic data initiatives (“Driver Projects”). We review how GA4GH is addressing the major areas in which genomics is currently deployed including rare disease, common disease, cancer, and infectious disease. Finally, we describe differences between genomic sequence data that are generated for research versus healthcare purposes, and define strategies for meeting the unique challenges of responsibly enabling access to data acquired in the clinical setting.

GA4GH organization

GA4GH has partnered with 24 real-world genomic data initiatives (Driver Projects) to ensure its standards are fit for purpose and driven by real-world needs. Driver Projects make a commitment to help guide GA4GH development efforts and pilot GA4GH standards (see Table 2). Each Driver Project is expected to dedicate at least two full-time equivalents to GA4GH standards development, which takes place in the context of GA4GH Work Streams (see Figure 1). Work Streams are the key production teams of GA4GH, tackling challenges in eight distinct areas across the data life cycle (see Box 1). Work Streams consist of experts from their respective sub-disciplines and include membership from Driver Projects as well as hundreds of other organizations across the international genomics and health community.

Figure thumbnail gr1
Figure 1Matrix structure of the Global Alliance for Genomics and HealthShow full caption


Box 1
GA4GH Work Stream focus areasThe GA4GH Work Streams are the key production teams of the organization. Each tackles a specific area in the data life cycle, as described below (URLs listed in the web resources).

  • (1)Data use & researcher identities: Develops ontologies and data models to streamline global access to datasets generated in any country9,10
  • (2)Genomic knowledge standards: Develops specifications and data models for exchanging genomic variant observations and knowledge18
  • (3)Cloud: Develops federated analysis approaches to support the statistical rigor needed to learn from large datasets
  • (4)Data privacy & security: Develops guidelines and recommendations to ensure identifiable genomic and phenotypic data remain appropriately secure without sacrificing their analytic potential
  • (5)Regulatory & ethics: Develops policies and recommendations for ensuring individual-level data are interoperable with existing norms and follow core ethical principles
  • (6)Discovery: Develops data models and APIs to make data findable, accessible, interoperable, and reusable (FAIR)
  • (7)Clinical & phenotypic data capture & exchange: Develops data models to ensure genomic data is most impactful through rich metadata collected in a standardized way
  • (8)Large-scale genomics: Develops APIs and file formats to ensure harmonized technological platforms can support large-scale computing

For more articles on Open Access, Science 2.0, and Data Networks for Genomics on this Open Access Scientific Journal see:

Scientific Curation Fostering Expert Networks and Open Innovation: Lessons from Clive Thompson and others

Icelandic Population Genomic Study Results by deCODE Genetics come to Fruition: Curation of Current genomic studies

eScientific Publishing a Case in Point: Evolution of Platform Architecture Methodologies and of Intellectual Property Development (Content Creation by Curation) Business Model 

UK Biobank Makes Available 200,000 whole genomes Open Access

Systems Biology Analysis of Transcription Networks, Artificial Intelligence, and High-End Computing Coming to Fruition in Personalized Oncology

Read Full Post »

UK Biobank Makes Available 200,000 whole genomes Open Access

Reporter: Stephen J. Williams, Ph.D.

The following is a summary of an article by Jocelyn Kaiser, published in the November 26, 2021 issue of the journal Science.

To see the full article please go to https://www.science.org/content/article/200-000-whole-genomes-made-available-biomedical-studies-uk-effort

The UK Biobank (UKBB) this week unveiled to scientists the entire genomes of 200,000 people who are part of a long-term British health study.

The trove of genomes, each linked to anonymized medical information, will allow biomedical scientists to scour the full 3 billion base pairs of human DNA for insights into the interplay of genes and health that could not be gleaned from partial sequences or scans of genome markers. “It is thrilling to see the release of this long-awaited resource,” says Stephen Glatt, a psychiatric geneticist at the State University of New York Upstate Medical University.

Other biobanks have also begun to compile vast numbers of whole genomes, 100,000 or more in some cases (see table, below). But UKBB stands out because it offers easy access to the genomic information, according to some of the more than 20,000 researchers in 90 countries who have signed up to use the data. “In terms of availability and data quality, [UKBB] surpasses all others,” says physician and statistician Omar Yaxmehen Bello-Chavolla of the National Institute for Geriatrics in Mexico City.

Enabling your vision to improve public health

Data drives discovery. We have curated a uniquely powerful biomedical database that can be accessed globally for public health research. Explore data from half a million UK Biobank participants to enable new discoveries to improve public health.

Data Showcase

Future data releases

This UKBB biobank represents genomes collected from 500,000 middle-age and elderly participants for 2006 to 2010. The genomes are mostly of a European descent. Other large scale genome sequencing ventures like Iceland’s DECODE, which collected over 100,000 genomes, is now a subsidiary of Amgen, and mostly behind IP protection, not Open Access as this database represents.

UK Biobank is a large-scale biomedical database and research resource, containing in-depth genetic and health information from half a million UK participants. The database is regularly augmented with additional data and is globally accessible to approved researchers undertaking vital research into the most common and life-threatening diseases. It is a major contributor to the advancement of modern medicine and treatment and has enabled several scientific discoveries that improve human health.

A summary of some large scale genome sequencing projects are show in the table below:

BiobankCompleted Whole GenomesRelease Information
UK Biobank200,000300,000 more in early 2023
TransOmics for
Precision Medicien
161,000NIH requires project
specific request
Million Veterans
Program
125,000Non-Veterans Affairs
researchers get first access
100,000 Genomes
Project
120,000Researchers must join Genomics
England collaboration
All of Us90,000NIH expects to release 2022

Other Related Articles on Genome Biobank Projects in this Open Access Online Scientific Journal Include the Following:

Icelandic Population Genomic Study Results by deCODE Genetics come to Fruition: Curation of Current genomic studies

Exome Aggregation Consortium (ExAC), generated the largest catalogue so far of variation in human protein-coding regions: Sequence data of 60,000 people, NOW is a publicly accessible database

Systems Biology Analysis of Transcription Networks, Artificial Intelligence, and High-End Computing Coming to Fruition in Personalized Oncology

Diversity and Health Disparity Issues Need to be Addressed for GWAS and Precision Medicine Studies

Read Full Post »

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

View all Tweet activity View Tweet activity

Top Follower followed by 7,598 people

Ryan Gravatt@gravatt FOLLOWS YOU

Christian, father, husband. Owner @RaconteurMC. Strategist for comms, digital. Former award-winning journalist. Proverbs 3:5-6 View profile

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

Tweet impressions

17.6K

Profile visits

861

Mentions

211

New followers

2

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

LPBI Group’s Logo
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

https://www.youtube.com/results?search_query=Disruptive+Dozen+2021+World+Medical+Innovation+Forum

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 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

1

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

1

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

1

2

You Retweeted

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

2

3

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

5

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

2

1

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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

1

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

1

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

2

You Retweeted

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

You Retweeted

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

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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

You Retweeted

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/

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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

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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

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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

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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

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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

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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

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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

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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

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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

2

2

You Retweeted

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)

1

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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

1

1

You Retweeted

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

3

3

You Retweeted

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

1

3

6

You Retweeted

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/

2

1

You Retweeted

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)

1

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You Retweeted

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

1

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You Retweeted

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

8

16

You Retweeted

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

6

11

You Retweeted

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

 

1

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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

2

6

You Retweeted

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

You Retweeted

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

1

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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

22

You Retweeted

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.

1

1

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…

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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

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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

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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”

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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

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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

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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

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#TUBiol5227: Biomarkers & Biotargets: Genetic Testing and Bioethics

Curator: Stephen J. Williams, Ph.D.

The advent of direct to consumer (DTC) genetic testing and the resultant rapid increase in its popularity as well as companies offering such services has created some urgent and unique bioethical challenges surrounding this niche in the marketplace. At first, most DTC companies like 23andMe and Ancestry.com offered non-clinical or non-FDA approved genetic testing as a way for consumers to draw casual inferences from their DNA sequence and existence of known genes that are linked to disease risk, or to get a glimpse of their familial background. However, many issues arose, including legal, privacy, medical, and bioethical issues. Below are some articles which will explain and discuss many of these problems associated with the DTC genetic testing market as well as some alternatives which may exist.

‘Direct-to-Consumer (DTC) Genetic Testing Market to hit USD 2.5 Bn by 2024’ by Global Market Insights

This post has the following link to the market analysis of the DTC market (https://www.gminsights.com/pressrelease/direct-to-consumer-dtc-genetic-testing-market). Below is the highlights of the report.

As you can see,this market segment appears to want to expand into the nutritional consulting business as well as targeted biomarkers for specific diseases.

Rising incidence of genetic disorders across the globe will augment the market growth

Increasing prevalence of genetic disorders will propel the demand for direct-to-consumer genetic testing and will augment industry growth over the projected timeline. Increasing cases of genetic diseases such as breast cancer, achondroplasia, colorectal cancer and other diseases have elevated the need for cost-effective and efficient genetic testing avenues in the healthcare market.
 

For instance, according to the World Cancer Research Fund (WCRF), in 2018, over 2 million new cases of cancer were diagnosed across the globe. Also, breast cancer is stated as the second most commonly occurring cancer. Availability of superior quality and advanced direct-to-consumer genetic testing has drastically reduced the mortality rates in people suffering from cancer by providing vigilant surveillance data even before the onset of the disease. Hence, the aforementioned factors will propel the direct-to-consumer genetic testing market overt the forecast timeline.
 

DTC Genetic Testing Market By Technology

Get more details on this report – Request Free Sample PDF
 

Nutrigenomic Testing will provide robust market growth

The nutrigenomic testing segment was valued over USD 220 million market value in 2019 and its market will witness a tremendous growth over 2020-2028. The growth of the market segment is attributed to increasing research activities related to nutritional aspects. Moreover, obesity is another major factor that will boost the demand for direct-to-consumer genetic testing market.
 

Nutrigenomics testing enables professionals to recommend nutritional guidance and personalized diet to obese people and help them to keep their weight under control while maintaining a healthy lifestyle. Hence, above mentioned factors are anticipated to augment the demand and adoption rate of direct-to-consumer genetic testing through 2028.
 

Browse key industry insights spread across 161 pages with 126 market data tables & 10 figures & charts from the report, “Direct-To-Consumer Genetic Testing Market Size By Test Type (Carrier Testing, Predictive Testing, Ancestry & Relationship Testing, Nutrigenomics Testing), By Distribution Channel (Online Platforms, Over-the-Counter), By Technology (Targeted Analysis, Single Nucleotide Polymorphism (SNP) Chips, Whole Genome Sequencing (WGS)), Industry Analysis Report, Regional Outlook, Application Potential, Price Trends, Competitive Market Share & Forecast, 2020 – 2028” in detail along with the table of contents:
https://www.gminsights.com/industry-analysis/direct-to-consumer-dtc-genetic-testing-market
 

Targeted analysis techniques will drive the market growth over the foreseeable future

Based on technology, the DTC genetic testing market is segmented into whole genome sequencing (WGS), targeted analysis, and single nucleotide polymorphism (SNP) chips. The targeted analysis market segment is projected to witness around 12% CAGR over the forecast period. The segmental growth is attributed to the recent advancements in genetic testing methods that has revolutionized the detection and characterization of genetic codes.
 

Targeted analysis is mainly utilized to determine any defects in genes that are responsible for a disorder or a disease. Also, growing demand for personalized medicine amongst the population suffering from genetic diseases will boost the demand for targeted analysis technology. As the technology is relatively cheaper, it is highly preferred method used in direct-to-consumer genetic testing procedures. These advantages of targeted analysis are expected to enhance the market growth over the foreseeable future.
 

Over-the-counter segment will experience a notable growth over the forecast period

The over-the-counter distribution channel is projected to witness around 11% CAGR through 2028. The segmental growth is attributed to the ease in purchasing a test kit for the consumers living in rural areas of developing countries. Consumers prefer over-the-counter distribution channel as they are directly examined by regulatory agencies making it safer to use, thereby driving the market growth over the forecast timeline.
 

Favorable regulations provide lucrative growth opportunities for direct-to-consumer genetic testing

Europe direct-to-consumer genetic testing market held around 26% share in 2019 and was valued at around USD 290 million. The regional growth is due to elevated government spending on healthcare to provide easy access to genetic testing avenues. Furthermore, European regulatory bodies are working on improving the regulations set on the direct-to-consumer genetic testing methods. Hence, the above-mentioned factors will play significant role in the market growth.
 

Focus of market players on introducing innovative direct-to-consumer genetic testing devices will offer several growth opportunities

Few of the eminent players operating in direct-to-consumer genetic testing market share include Ancestry, Color Genomics, Living DNA, Mapmygenome, Easy DNA, FamilytreeDNA (Gene By Gene), Full Genome Corporation, Helix OpCo LLC, Identigene, Karmagenes, MyHeritage, Pathway genomics, Genesis Healthcare, and 23andMe. These market players have undertaken various business strategies to enhance their financial stability and help them evolve as leading companies in the direct-to-consumer genetic testing industry.
 

For example, in November 2018, Helix launched a new genetic testing product, DNA discovery kit, that allows customer to delve into their ancestry. This development expanded the firm’s product portfolio, thereby propelling industry growth in the market.

The following posts discuss bioethical issues related to genetic testing and personalized medicine from a clinicians and scientisit’s perspective

Question: Each of these articles discusses certain bioethical issues although focuses on personalized medicine and treatment. Given your understanding of the robust process involved in validating clinical biomarkers and the current state of the DTC market, how could DTC testing results misinform patients and create mistrust in the physician-patient relationship?

Personalized Medicine, Omics, and Health Disparities in Cancer:  Can Personalized Medicine Help Reduce the Disparity Problem?

Diversity and Health Disparity Issues Need to be Addressed for GWAS and Precision Medicine Studies

Genomics & Ethics: DNA Fragments are Products of Nature or Patentable Genes?

The following posts discuss the bioethical concerns of genetic testing from a patient’s perspective:

Ethics Behind Genetic Testing in Breast Cancer: A Webinar by Laura Carfang of survivingbreastcancer.org

Ethical Concerns in Personalized Medicine: BRCA1/2 Testing in Minors and Communication of Breast Cancer Risk

23andMe Product can be obtained for Free from a new app called Genes for Good: UMich’s Facebook-based Genomics Project

Question: If you are developing a targeted treatment with a companion diagnostic, what bioethical concerns would you address during the drug development process to ensure fair, equitable and ethical treatment of all patients, in trials as well as post market?

Articles on Genetic Testing, Companion Diagnostics and Regulatory Mechanisms

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges

New York Times vs. Personalized Medicine? PMC President: Times’ Critique of Streamlined Regulatory Approval for Personalized Treatments ‘Ignores Promising Implications’ of Field

Live Conference Coverage @Medcitynews Converge 2018 Philadelphia: Early Diagnosis Through Predictive Biomarkers, NonInvasive Testing

Protecting Your Biotech IP and Market Strategy: Notes from Life Sciences Collaborative 2015 Meeting

Question: What type of regulatory concerns should one have during the drug development process in regards to use of biomarker testing? From the last article on Protecting Your IP how important is it, as a drug developer, to involve all payers during the drug development process?

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New avenues for research in membrane biology reveals the mobility of protein at work

Curator and Reporter: Dr. Premalata Pati, Ph.D., Postdoc

Membrane proteins (MPs) are proteins that exist in the plasma membrane and conduct a variety of biological functions such as ion transport, substrate transport, and signal transduction. MPs undergo function-related conformational changes on time intervals spanning from nanoseconds to seconds. Many MP structures have been solved thanks to recent developments in structural biology, particularly in single-particle cryo-Electron Microscopy (cryo-EM). Obtaining time-resolved dynamic information on MPs in their membrane surroundings, on the other hand, remains a significant difficulty.

OmpG (Open state) in a fully hydrated dimyristoylphosphatidylcholine (DMPC) bilayer. The protein is shown in light green cartoon. Lipids units are depicted in yellow, while their phosphate and choline groups are illustrated as orange and green van der Waals spheres, respectively. Potassium and chloride counterions are shown in green and purple, respectively. A continuous and semi-transparent cyan representation is used for water.
https://static-content.springer.com/esm/art%3A10.1038%2Fs41467-021-24660-1/MediaObjects/41467_2021_24660_MOESM1_ESM.pdf

Weill Cornell Medicine (WCM) researchers have found that they can record high-speed protein movements while linking them to function. The accomplishment should allow scientists to examine proteins in more depth than ever before, and in theory, it should allow for the development of drugs that work better by hitting their protein targets much more effectively.

The researchers utilized High-Speed Atomic Force Microscopy (HS-AFM) to record the rapid motions of a channel protein and published in a report in Nature Communications on July 16. Such proteins generally create channel or tube-like structures in cell membranes, which open to allow molecules to flow under particular conditions. The researchers were able to record the channel protein’s rapid openings and closings with the same temporal resolution as single channel recordings, a typical technique for recording the intermittent passage of charged molecules through the channel.

Senior author Simon Scheuring, professor of physiology and biophysics in anesthesiology at WCM, said,

There has been a significant need for a tool like this that achieves such a high bandwidth that it can ‘see’ the structural variations of molecules as they work.

Researchers can now produce incredibly detailed photographs of molecules using techniques like X-ray crystallography and electron microscopy, showing their structures down to the atomic scale. The average or dominant structural positionings, or conformations, of the molecules, are depicted in these “images,” which are often calculated from thousands of individual photos. In that way, they’re similar to the long-exposure still photos from the dawn of photography.

Many molecules, on the other hand, are flexible and always-moving machinery rather than fixed structures. Scientists need to generate videos, not still photos, to reveal how such molecules move as they work, to see how their motion translates to function to catch their critical functional conformations, which may only exist for a brief moment. Current techniques for dynamic structural imaging, on the other hand, have several drawbacks, one of which being the requirement for fluorescent tags to be inserted on the molecules being photographed in many cases.

Scheuring and his lab were early adopters of the tag-free HS-AFM approach for studying molecular dynamics. The technology, which can photograph molecules in a liquid solution similar to a genuine cellular environment, employs an extremely sensitive probe, similar to a record player’s stylus, to feel its way over a molecule and therefore build up a picture of its structure. Standard HS-AFM isn’t quick enough to capture the high-speed dynamics of many proteins, but Scheuring and colleagues have developed a modified version, HS-AFM height spectroscopy (HS-AFM-HS), that works much faster by collecting dynamic changes in only one dimension: height.

The researchers used HS-AFM-HS to record the opening and closing of a relatively simple channel protein, OmpG, found in bacteria and widely studied as a model channel protein in the new study, led by the first author Raghavendar Reddy Sanganna Gari, a postdoctoral research associate in Scheuring’s laboratory. They were able to monitor OmpG gating at an effective rate of roughly 20,000 data points per second, seeing how it transitioned from open to closed states or vice versa as the acidity of the surrounding fluid varied.

More significantly, they were able to correlate structural dynamics with functional dynamics in a membrane protein of this size for the first time in a partnership with Crina Nimigean, professor of physiology and biophysics in anesthesiology, and her group at WCM.

The demonstration opens the door for a wider application of this method in basic biology and drug development.

Sanganna Gari stated,

We’re now in an exciting period of HS-AFM technology, for example using this technique to study how some drugs modulate the structural dynamics of the channel proteins they target.

Main Source

Technique reveals proteins moving as they work. By Jim Schnabel in Cornell Chronicle, August 16, 2021.

https://news.cornell.edu/stories/2021/08/technique-reveals-proteins-moving-they-work

Other Related Articles published in this Open Access Online Scientific Journal include the following:

Cryo-EM disclosed how the D614G mutation changes SARS-CoV-2 spike protein structure.

Reporter: Dr. Premalata Pati, Ph.D., Postdoc

https://pharmaceuticalintelligence.com/2021/04/10/cryo-em-disclosed-how-the-d614g-mutation-changes-sars-cov-2-spike-protein-structure/

Proteins, Imaging and Therapeutics

Larry H Bernstein, MD, FCAP, Curator, LPBI

https://pharmaceuticalintelligence.com/2015/10/01/proteins-imaging-and-therapeutics/

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

Curator: Stephen J. Williams, PhD

https://pharmaceuticalintelligence.com/2021/07/19/from-high-throughput-assay-to-systems-biology-new-tools-for-drug-discovery/

Imaging break-through: Fusion of microscopy and mass spectrometry produces detailed map of protein distribution

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2015/03/18/imaging-break-through-fusion-of-microscopy-and-mass-spectrometry-produces-detailed-map-of-protein-distribution/

Advanced Microscopic Imaging

Larry H Bernstein, MD, FCAP, Curator, LPBI

https://pharmaceuticalintelligence.com/2016/02/07/advanced-microscopic-imaging/

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