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CRISPR – The Business and Legal Aspects of IP Development, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Patent on Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription was awarded to UC, Berkeley on October 30, 2018
site-specific modification of a target DNA and/or a polypeptide associated with the target DNA, a DNA-targeting RNA
genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.
Reporter: Aviva Lev-Ari, PhD, RN
( 1of1 )
United States Patent
10,113,167
Doudna , et al.
October 30, 2018
Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
AbstractThe present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.
Inventors:
Doudna; Jennifer A. (Berkeley, CA), Jinek; Martin (Berkeley, CA), Chylinski; Krzysztof (Vienna, AT), Charpentier; Emmanuelle (Braunschweig, DE)
Applicant:
Name
City
State
Country
Type
The Regents of the University of California
University of Vienna
Charpentier; Emmanuelle
Oakland
Vienna
Braunschweig
CA
N/A
N/A
US
AT
DE
Assignee:
The Regents of the University of California (Oakland, CA) University of Vienna (Vienna, AT) Charpentier; Emmanuelle (Braunschweig, DE)
UC Berkeley team awarded second CRISPR-Cas9 patent
“Today’s news … represents yet another validation of the historic and field-changing breakthrough invented by scientists Jennifer Doudna, Emmanuelle Charpentier, and their team… The patent announced today specifically highlights the CRISPR-Cas9 invention’s ability to edit DNA in any setting, including within animal and human cells. It also highlights its utility in several formats across both dual-RNA and single-RNA configurations, useful for therapy for genetic diseases and for improving food security.”
— Edward Penhoet, special adviser to the UC Berkeley chancellor, tells Axios
The details: According to the patent, the compositions can be used in animal or human cells, and can work as either 2 separate pieces of RNA or a single piece of RNA.
Penhoet says the new patent covers 2 RNA components that together form the “DNA-targeting-RNA,” with one that targets the particular sequence of DNA needed to be edited and the other that binds with the Cas9 protein.
This follows another patent given to UC Berkeley in June on methods to use CRISPR-cas9.
The patents cover the composites used by CRISPR-Cas9 within human, plant, animal and bacteria cells.
Both allow the use of strands of RNA “that can be shorter than naturally-occurring RNA components. This allows them to be more easily used and, therefore, is a form often preferred,” Penhoet says.
Will the Supreme Court accept a UC Berkeley Appeal of the Sep. 10th, US Court of Appeals for the Federal Circuit decision to uphold the patent filed by the Broad Institute on CRISPR/Cas9 gene editing?, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Will the Supreme Court accept a UC Berkeley Appeal of the Sep. 10th, US Court of Appeals for the Federal Circuit decision to uphold the patent filed by the Broad Institute on CRISPR/Cas9 gene editing?
Reporter: Aviva Lev-Ari, PhD, RN
UPDATED on June 6, 2019
Several companies were founded on the initial CRISPR IP rights granted to various individuals and institutions. These companies include Intellia Therapeutics and its parent company, Caribou Biosciences (Berkeley), CRISPR Therapeutics and ERS Genomics (Emmanuelle Charpentier), and Editas Medicine (Broad) as well as the Broad Institute itself. Anyone aiming to commercialize CRISPR technology must obtain licenses from one or more of these companies. However, Broad and Berkeley have followed the long-standing recommendations that federally funded academic institutions grant non-exclusive licenses to university researchers and nonprofits.
If all of that weren’t complex enough, there are certain overlaps between the patents. For example, Editas, CRISPR Therapeutics, and Intellia all offer licenses to treat human diseases. But ERS Genomics specifically excludes a therapeutics option. Meanwhile, both Editas and Intellia offer licenses for stem cells, CAR-T cells, and Alpha-1 antitrypsin while Caribou Biosciences and the Broad Institute do not. In short, navigating the CRISPR IP thicket can be extremely confusing. And, unfortunately, it is likely to become even more so.
While Berkeley’s notice of allowance does help put out the flames, until recently, most of the fights have centered on the Cas9 protein. But, in the last several years, research has shown that the CRISPR-Cpf1 protein, also known as Cas12a, is potentially more effective than Cas9. Companies like Mammoth Biosciences have already been founded off of Cas12a technology. Patents involving the Cas12a-RNA complex are already pending on behalf of Berkeley and the Broad Institute.
On 2018, Sep. 10th, the US Court of Appeals for the Federal Circuit agreed to uphold the patent filed by the Broad Institute on CRISPR/Cas9 gene editing in organisms with complex cells – UC Berkeley team can appeal this decision to the US Supreme Court, it is unclear whether the Supreme Court will accept this case.
According to Appeal and Interference Statistics 11/30/2016
In recent years, more than half of PTAB’s decisions have been upheld. “The Federal Circuit heard three appeals of interferences in 2016,” said intellectual property expert Jacob Sherkow of New York Law School. “All three were at least affirmed in part. It’s completely unclear whether that’s meaningful — it’s an N of 3–but there you go.” Overall, on 155 appeals since PTAB was created in 2012, the Federal Circuit affirmed 120 on every issue, dismissed or reversed 21 on every issue, and issued partial decisions (that is, upholding parts of a PTAB decision and reversing others) in the other 14.
SOURCE
Disputed CRISPR Patents Stay with Broad Institute, U.S. Panel Rules
Sickle Cell and Beta Thalassemia chosen for first human trial of the gene editing technology, CRISPR by sponsoring companies CRISPR Therapeutics and Vertex Pharmaceuticals, trial at a single site in Germany, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Sickle Cell and Beta Thalassemia chosen for first human trial of the gene editing technology, CRISPR by sponsoring companies CRISPR Therapeutics and Vertex Pharmaceuticals, trial at a single site in Germany,
Reporter: Aviva Lev-Ari, PhD, RN
UPDATED on 3/09/2019
CRISPR Therapeutics share up on announcement of first dosing in the joint Vertex sponsored trial for their gene editing therapy CTX001 for patients with beta thalassemia.
CRISPR Therapeutics (CRSP – Free Report) and its partner Vertex Pharmaceuticals (VRTX – Free Report) announced the dosing of the first patient in a phase I/II study evaluating the CRISPR/Cas9 gene-editing therapy, CTX001, in patients with beta thalassemia, a form of anemia. This is the first in-human use of CTX001 in any clinical study.
The companies also enrolled first patients in another phase I/II study evaluating CTX001 in patients with severe sickle cell disease (“SCD”), a severe hereditary form of anemia. Dosing in the study is expected to start in mid-2019.
Shares of CRISPR Therapeutics surged more than 25% following the announcement of the progress made by the company in studies on CTX001. However, the stock has declined 14.3% in the past year.
We remind investors that last month, the FDA assigned Fast Track designation CTX001 for the treatment of SCD. With this designation, the drug is expected to be granted a priority review once the company files a new drug application.
In December 2018, Vertex and CRISPR Therapeutics selected CTX001 to move into clinical development as a gene edited treatment for sickle cell disease and beta -thalassemia. CTX001 has been developed using CRISPR Therapeutics’ proprietary CRISPR/Cas9 technology. The companies had entered into a strategic research collaboration in 2015 to co-develop and co-commercialize CTX001. Per the agreement, they will equally share all R&D costs and profits worldwide. Vertex has rights to license up to six new gene editing treatments (including CTX001), developed using the CRISPR/Cas9 technology from CRISPR that will emerge from the joint research deal.
Other than CRISPR Therapeutics, Intellia Therapeutics (NTLA – Free Report) and Editas Medicine, Inc (EDIT – Free Report) plan to carry out clinical studies using CRISPR Cas9 to cure diseases.
CRISPR Therapeutics also announced its fourth-quarter results in a separate press release. The company reported revenues of $0.1 million, which came from collaborations, compared to $32.3 million in year-ago period. Reported loss was 92 cents per share in the fourth quarter. The company achieved breakeven results in the year-ago quarter.
The company remains on track to initiate an immuno-oncology study in the first half of 2019 on its CAR-T cell therapy candidate, CTX110, for treating CD19+ malignancies. The company is the sole owner of the candidate. Since September, the company has inked or modified several collaboration agreements with other pharma companies for pre-clinical development of its new CRISPR/Cas9 gene editing candidates.
NIH launches initiative to accelerate genetic therapies to cure sickle cell disease
“Our scientific investments have brought us to a point where we have many tools available to correct or compensate for the defective gene that causes sickle cell disease. We are now ready to use these tools to speed up our quest for a cure,” said Gary H. Gibbons, M.D., director of NIH’s National Heart, Lung, and Blood Institute (NHLBI), which is leading the effort.
Vertex licensed CTX001, an autologous gene-edited hematopoietic stem cell therapy, from CRISPR in December. It was the first CRISPR-based treatment to come out of a four-year, $105 million deal the pair struck in 2015. At the time, Vertex paid up $75 million in cash and took a $30 million stake in CRISPR Therapeutics in exchange for the right to license up to six gene-editing programs. CTX001 is being developed for the blood disorders sickle cell disease and beta thalassemia.
Both disorders are caused by mutations in the beta-globin gene, which codes for a part of hemoglobin, the oxygen-carrying component of red blood cells. This results in missing or defective hemoglobin. CTX001 was developed on the knowledge that fetal hemoglobin—found in newborn babies but later replaced by adult hemoglobin—can be protective in adults who have blood disorders.
CTX001 uses CRISPR gene-editing ex vivo—that is, outside the body. A patient’s cells are harvested and edited to increase fetal hemoglobin levels in the patient’s blood cells. The edited cells are then infused back into the patient where they are expected to produce blood cells with fetal hemoglobin and compensate for defective adult hemoglobin.
To develop new tissues, researchers at the Medical Research Council Center for Regenerative Medicine at the University of Edinburgh have found that stem cells transformed into 3-D liver tissue can support liver function when implanted into the mice suffering with a liver disease.
The scientists stimulated human embryonic stem cells and induced pluripotent stem cells to mature pluripotent stem cells into liver cells, hepatocytes. Hepatocytes are the chief functional cells of the liver and perform an astonishing number of metabolic, endocrine and secretory functions. Hepatocytes are exceptionally active in synthesis of protein and lipids for export. The cells are grown in 3-D conditions as small spheres for over a year. However, keeping the stem cells as liver cells for a long time is very difficult, because the viability of hepatocytes decreases in-vitro conditions.
Succeeding the discovery, the team up with materials chemists and engineers to detect appropriate polymers that have already been approved for human use that can be developed into 3-D scaffolds. The best material to use a biodegradable polyester, called polycaprolactone (PCL).PCL is degraded by hydrolysis of its ester linkages in physiological conditions (such as in the human body) and it is especially interesting for the preparation of long term implantable devices, owing to its degradation which is even slower than that of polylactide. They spun the PCL into microscopic fibers that formed a scaffold one centimeter square and a few millimeters thick.
At the same time, hepatocytes derived from embryonic cells had been grown in culture for 20 days and were then loaded onto the scaffolds and implanted under the skin of mice.Blood vessels successfully grew on the scaffolds with the mice having human liver proteins in their blood, demonstrating that the tissue had successfully integrated with the circulatory system. The scaffolds were not rejected by the animals’ immune systems.
The scientists tested the liver tissue scaffolds in mice with tyrosinaemia,a potentially fatal genetic disorder where the enzymes in the liver that break down the amino acid tyrosine are defective, resulting in the accumulation of toxic metabolic products. The implanted liver tissue aided the mice with tyrosinaemia to break down tyrosine and the mice finally lost less weight, had less buildup of toxins in the blood and exhibited fewer signs of liver damage than the control group that received empty scaffolds.
According to Rob Buckle, PhD, Chief Science Officer at the MRC, “Showing that such stem cell-derived tissue is able to reproduce aspects of liver function in the lab also offers real potential to improve the testing of new drugs where more accurate models of human tissue are needed”. It is believed that the discovery could be the next step towards harnessing stem cell reprogramming technologies to provide renewable supplies of liver tissue products for transplantation.
New CRISPR Approach Transforms Skin Cells into Pluripotent Stem Cells
Reporter: Irina Robu, PhD
Dr. Timo Otonkoski, University of Helsinki and Dr.Juha Kere, King’s College London succeeded on reprograming skin cells into pluripotent stem cells by activating cell’s own genes using gene editing technology, CRISPR-Cas9-based gene activation (CRISPRa) that can be used to activate genes. The method uses a blunt version of Cas9 ‘gene scissors’ that does not cut DNA and can consequently be used to activate gene expression without mutating the genome. Previously, reprogramming was only possible by artificially introducing the critical transformation genes known as Yamanaka Factors into skin cells where they are normally inactive.
According to a study that is published in Nature Communication, called Human Pluripotent Reprogramming with CRISPR activators which show that CRISPRa is an attractive tool for cellular reprogramming applications due to its high multiplex capacity and direct alignment of endogenous loci. In the article, it is presented that reprogramming of primary human dermal fibroblasts to induced pluripotent stem cells with CRISPRa, the aimed at endogenous cells. The data shows that human body cells can only be reprogrammed into iPS cells with CRISPRa, and the findings reveal the involvement of EEA motif-associated mechanisms in cellular reprogramming.
The discovery also advocates that it might be likely to improve many other reprogramming tasks by addressing genetic elements that are typical of the intended target cell type. According to Jere Weltner, PhD student working on the project “the technology can find practical application in biobanking and many other applications of tissue technology.
On June 12, 2018 – Berkeley was granted a patent on using CRISPR/Cas9 to edit single-stranded RNA. On June 19, 2018 – Berkeley was granted a second patent, covering the use of CRISPR-Cas9 gene editing with formats that will be particularly useful in developing human therapeutics and improvements in food security, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
On June 12, 2018 – Berkeley was granted a patent on using CRISPR/Cas9 to edit single-stranded RNA. On June 19, 2018 – Berkeley was granted a second patent, covering the use of CRISPR-Cas9 gene editing with formats that will be particularly useful in developing human therapeutics and improvements in food security.
Reporter and Curator: Aviva Lev-Ari, PhD, RN
UPDATED on 6/6/2019
What is the future of CRISPR IP rights?
Most likely, a complex one. Genomic breakthroughs are on the rise as CRISPR unlocks the secrets of the human genome. The CRISPR-Cas9 IP battle seems settled for now. And there is no indication that Cas12a will kick up nearly the same kinds of patent fights. But that is no guarantee for the future. With a potential worth of billions of dollars, CRISPR has the capability to break scientific partnerships as easily as it does DNA.
The best way to stay out of the IP rabbit hole is to use CRISPR for research or non-profit purposes. But even commercial companies looking to build CRISPR-based platforms should carefully review the relevant licenses from each institution, especially as there are often non-exclusive options for certain kinds of CRISPR products and services. Unsurprisingly, the most complex CRISPR IP rights seem to center around therapeutics. But other industries including agriculture should be clear on their licensing options as well.
We are still in the early days of CRISPR technology and there is a lot to learn both in and out of the lab. The hope is that licensing CRISPR will become more straightforward or at least clearer and more organized. Synthego CSO Rich Stoner spoke with SynBioBeta’s Kevin Costa about the current atmosphere surrounding CRISPR IP rights. “Yes, there’s a lot of litigation and contention around the core patents. But the pace of innovation to create new nucleases, new ways to edit and more predictability means that we’re very optimistic about the future of the technologies we’re deploying now as well as over the next few years.”
But that future remains a little way off—the dust is still settling.
Patent involved in interference proceedings will add to university’s gene-editing portfolio
The U.S. Patent and Trademark Office has issued a notice of allowance for a University of California patent application covering systems and methods for using single molecule guide RNAs that, when combined with the Cas9 protein, create more efficient and effective ways for scientists to target and edit genes. U.S. patent application number 13/842,859, which had notably been examined in advance of a prior interference proceeding involving the Broad Institute, specifically focuses on methods and systems for modifying a target DNA molecule in any setting, both in vitro and within live cells, using one or multiple single guide RNAs, across every cell type. The associated patent is expected to issue in the next 6-9 weeks.
This CRISPR-Cas9 DNA-targeting technology, invented by Jennifer Doudna and Martin Jinek of the University of California, Berkeley, along with Emmanuelle Charpentier at Umea University and Krzystof Chylinski at the University of Vienna, is a fundamental molecular tool for editing genes. Together, this patent application and prior U.S. Patent Numbers 10,000,772 and 10,113,167, cover CRISPR-Cas9 methods and compositions useful as gene-editing scissors in any setting, including in vitro, as well as within live plant, animal and human cells.
“We are pleased the patent application is now allowed and that the issued patent will encompass the use of CRISPR-Cas9 technology in any cellular or non-cellular environment. We expect to see continued momentum in the expansion of UC’s CRISPR patent portfolio in the coming months,” said Eldora L. Ellison, Ph.D., lead patent strategist on CRISPR matters for the University of California and a director at Sterne, Kessler, Goldstein & Fox. “The steadfast protection of the CRISPR intellectual property pioneered by the Doudna-Charpentier team is wholly focused on the improvement of human welfare.”
The patent covers methods of using optimized guide RNA formats (including single guide and dual guide formats) in certain environments, including eukaryotic cells (such as human, animal and plant cells). The optimized formats modify the part of a guide RNA that interacts with the CRISPR/Cas9 nuclease.
Who is it that deserves credit for turning a bacterial immune system into a revolutionary gene editing tool?
We suggest that it is as follows: Two owners of IP in Red
Gene Editing Consortium of Biotech Companies: CRISPR Therapeutics $CRSP, Intellia Therapeutics $NTLA, Caribou Biosciences, ERS Genomics, UC, Berkeley (Doudna’s IP) and University of Vienna (Charpentier’s IP), is appealing the decision ruled that there was no interference between the two sides, to the U.S. Court of Appeals for the Federal Circuit, targeting patents from The Broad Institute.
Patents for the wide use of CRISPR-Cas9 for gene editing all types of cells have already been issued to the Doudna-Charpentier team by the European Patent Office (representing more than 30 countries), the United Kingdom, China, Japan, Australia, New Zealand, Mexico and other countries. The scope of the United States patent issued today broadly includes the use of a CRISPR-Cas9 compound that is specially engineered to be more easily employed inside any type of plant or animal cell, or outside a cell, in order to modify a gene or the expression of a gene.
CRISPR Therapeutics, Intellia Therapeutics and Caribou Biosciences issued the following joint statement on the grant of the ‘772 patent:
“We believe that the U.S. patent ‘772 granted today covers the use of CRISPR/Cas9 genome editing with the RNA guide formats that are widely used throughout the industry. We anticipate this is the first of many patents that will be granted to UC on this foundational CRISPR/Cas9 intellectual property.”
In addition to this granted U.S. patent, applications from this patent estate have been found allowable in the United States and also have issued in Europe, the United Kingdom, China, Japan and various other countries worldwide. These patents cover the dual- and single-guide RNA compositions of the widely adopted CRISPR/Cas9 genome editing technology and their uses in all environments, including plant, animal and human cells as well as for use in human therapeutics.
Schematic representation of the CRISPR-Cas9 system. The Cas9 enzyme (orange) cuts the DNA (blue) in the location selected by the RNA (red). Image courtesy of Carlos Clarivan/Science Photo Library/NTB Scanpix
“Today’s patent is one of many we anticipate will be awarded to these inventors for their CRISPR-Cas9 invention,” said Edward Penhoet, special advisor to the UC Berkeley chancellor and special assistant to the University of California president. “Six years ago, the Doudna-Charpentier team was the first to file a patent application and publish on the necessary and sufficient components that enable CRISPR-Cas9 to be employed in all environments, including plant and animal cells. Their remarkable research has only accelerated since then, creating new jobs and opening up new possibilities to improve life.”
The U.S. patent granted today (10,000,772) is not involved in any interference proceeding before the Patent Trial and Appeal Board of the U.S. Patent and Trademark Office, or any appeal before the U.S. Court of Appeals for the Federal Circuit. The ‘772 patent is not impacted by the USPTO’s decision to terminate an interference between a separate UC patent application and a patent application owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology without reaching a decision on which inventors were the first to invent the use of CRISPR/Cas9 technology for genome editing. UC’s appeal of that decision was heard on April 30, 2018 by the U.S. Court of Appeals for the Federal Circuit, which will issue a decision in the future.
Comments made on On June 12, 2018 – Berkeley was granted a patent on using CRISPR/Cas9 to edit single-stranded RNA. On June 19, 2018 – Berkeley was granted a second patent, covering the use of CRISPR-Cas9 gene editing with formats
There have also been others commenting on the decision, including Jacob Sherkow, who’s an associate professor from the New York Law School. He said that he expected the second patent, in particular, to have “pretty minimal commercial value”. While former molecular biologist and biotech patent lawyer, Dr. Kevin Noonan have reportedly said he thinks UC Berkeley “is just happy to get a patent”.
In the never-ending saga of CRISPR patents, the University of California has finally put some points on the board in the U.S., with the Patent and Trademark Office granting it two genome-editing patents. One, granted on Tuesday, was first applied for in 2014. The other and more significant patent, applied for in 2015 but based on a 2012 discovery, will be granted next week.
The granted patent, number 9,994.831, covers “methods and compositions for modifying a single stranded target nucleic acid.” Next week’s, which is to be issued on June 19, covers the use of CRISPR-Cas9 for genome-editing in anything other than a bacterial cell and, specifically, where the guide RNA is formed by two other molecules “to form a total of 10 to 15 base pairs.” (Base pairs, or nucleotides, are the “letters” that constitute DNA and its cousin RNA.)
Original Tweets Re-Tweets and Likes by @pharma_BI and @AVIVA1950 at #kisymposium for 17th annual Summer Symposium: Breakthrough Cancer Nanotechnologies: Koch Institute, MIT Kresge Auditorium, June 15, 2018, 9AM-4PM
2.1.3.6 Original Tweets Re-Tweets and Likes by @pharma_BI and @AVIVA1950 at #kisymposium for 17th annual Summer Symposium: Breakthrough Cancer Nanotechnologies: Koch Institute, MIT Kresge Auditorium, June 15, 2018, 9AM-4PM, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Awesome interview with @NoubarAf. Can’t wait to see him on our panel at next week’s #KISymposium@kochinstitute@KI_Nanomedicine@MIThttps://twitter.com/MrJeffKarp/status/1005176997063086081…
SG: First micro bubble targeting KDR for human cancer detection to assess clinical safety in breast and ovarian lesions and feasibility to detect via ultrasound. Molecular imaging to histology #kisymposium
RW: Describing extracellular vessicles which are shed from cancer cells, contain RNA and Perrin reflecting cell of origin and the analysis tools to study #kisymposium
AB: amazing to see data on using the M13 phage with carbon nanotube homing to tumors prior to surgery allowing localization of tumors <1mm in size! #kisymposium
Great visualization in Angie Belcher’s presentation
Aviva Lev-Ari added,
Anne E Deconinck@AEDeconinck
Angie Belcher @kochinstitute@KI_Nanomedicine@MIT and her model of bacteriophage for detecting tiny tumors (<2mm) #KISymposiumhttps://twitter.com/milospm1206/status/1007627821634740224…
DA: wow, fascinating delivery of gene editing machinery to liver or lungs to correct gene defects, AAV for sgRNA and repair DNA plus nanoparticle containing Cas9 mRNA – and it worked in animal model to correct metabolic defect. #Kisymposium
Sangeeta Bhatia stresses the importance of #nano for early detection of #cancer especially in low-resource setting. Also promoting the launch of #MITnano this year #KIsymposium
Director of @mit nano Vladimir Bulovic says that nanoscale technology will advance every area of research at MIT through scientific collaboration. Looking forward to the discoveries it will enable! @kochinstitute …
Koch Institute at MITVerified account@kochinstitute
Holography and nanoplasmodics are just two examples of what the @WeisslederLab is working on to make cancer detection and diagnosis more accurate, affordable, and accessible, not to mention less invasive. Stay tuned for #KIsymposium videos later this summer!
My first panel since #BIO2018 and VERY glad it had diverse members and opinions. It was better as a result! Thanks to @kochinstitute and my co-panelists for a great discussion! #KIsymposiumhttps://twitter.com/kochinstitute/status/1007686581753405440…
Super excited about today’s #kisymposium panel discussion w/ @JMaraganore@Alnylam, @NoubarAf@FlagshipPioneer, Paula Hammond & Bob Langer @kochinstitute, Rijcken @cristaltherap, Bradbury …
IP and patents really matter for translation, valley of death still a problem, but look @MIT & @Stanford: Not about short term gains or royalties. It’s about IMPACT. Bob Langer @kochinstitute (doing good in the world) #kisymposium
Koch Institute at MITVerified account@kochinstitute
Time to kick off Session III: Nanosystems & Devices with @snbhatia, director of @KI_Nanomedicine. “Protease Nanosensors for Cancer Detection, Classification and Monitoring” Sounds like a sensitive subject! …
Paula Hammond: One of my main roles as a PI @kochinstitute is creating an environment in which creativity can thrive, and new ideas result in new platforms & new solutions #kisymposium
Koch Institute at MITVerified account@kochinstitute
Might need a tissue for this next talk, but that’s ‘cule. All will be (micro)well. Rashid Bashir from @eceILLINOIS presents “Micro and Nanotechnologies for Analysis of Tissues and Molecules” So fab! #KIsymposium
Na no na no, na no na no, hey hey, goodbye! Thanks to @KI_Nanomedicine, our amazing speakers, our wonderful sponsors, and everyone who came out to learn about Breakthrough Cancer Nanotechnologies at #KIsymposium 2018. See you on June 14, 2019 for AI/machine learning in cancer!
Koch Institute at MITVerified account@kochinstitute
Vladimir Bulović, founding director of #MITnano, presents “Nanoscale Discoveries for Transformative Breakthroughs” in the final session of #KIsymposium 2018. Through the ages, the lesson is clear: Dream…
This next one’s going to get personal. James Heath of @ISBUSA is taking “A Molecular View of Immuno-Oncology” with insight on how to personalize the field. We’re T-sold! (past tense of T-cell) #KIsymposium
Angela Belcher (@MITdeptofBE@KI_Nanomedicine) is sharing the stage with her portable, light-up bacteriophage to share “New Approaches for Finding Tiny Tumors: Towards Early Detection and Treatment of #ovariancancer” Ready, set, glow! #KIsymposium
Attending the 17th annual Cancer research symposium organized by @kochinstitute. Interesting talk by Angela Belcher on finding tiny tumors. #KIsymposium@MIT
(gene) Silence, everyone! It’s time for @KI_Nanomedicine member Dan Anderson (@MITChemE) to tell us about “Nanoparticle Formulations for RNA Therapy and Gene Editing.” #KIsymposium
@ben_ouyang and @TheChanLab taught me the importance of TAMs in tumour growth and NP delivery. The peptide developed by Suzie Pun that specifically targets M2 Mø has the potential to increase the efficiency of cancer nanotherapies #KIsymposium
Session II: Therapeutics continues with Suzie Pun (@UW researcher and @KI_Nanomedicine scientific advisor) presenting on “Modulating Tumor-Associated Macrophages.” (Wait, was that a “macro” in the title? We thought #Kisymposium was all nano all the time this year!)
S.S. Gambhir from @StanfordMed uses ultrasound imaging of nanobubbles (with gas core and active targeting) to image cancer at molecular level. Q: Do changes in pressure in body affect quality of imaging? #KIsymposium
Heads up! @Caltech‘s Mark Davis is talking about “Designing Nanoparticles to Safely Cross the Blood-Brain Barrier for Treating Brain Cancers” #KIsymposium
Yup, definite “props” to Angie for a dynamic engaging talk. Good science too. Very promising technology, data. Lots to discuss during the break. #KIsymposium
MD: Now moving into combo delivery with nanoparticle. Topo I and PARP toxicity so high when delivered in drug form, toxicity so high dosing had to be reduced to sub-efficacious levels. Now NCI study using delivery with nanoparticles to avoid tox #KISymposium
Great talk from Prof. Weissleder on the development of cancer diagnostics with actionable consequences @harvardmed @WeisslederLab @kochinstitute @EBioMedicine
Our first speaker, Sanjiv Sam Gambhir @Stanford talks Bubble Based Nanodiagnostics. Early detection = Prevention = deserves intense focus so we can save lives #kisymposium
2.1.5.3 National Academy of Sciences for work in chemical sciences: Jennifer Doudna, University of California, Berkeley, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Prasad Raghavendra and Jennifer Doudna received awards this week from the National Academy of Sciences for work incomputer science and chemical sciences, respectively.
Prasad Raghavendra, an associate professor of electrical engineering and computer science, and Jennifer Doudna, a professor of molecular and cell biology and of chemistry, were honored this week by the National Academy of Sciences for their innovative body of research.
Raghavendra shared the inaugural Michael and Sheila Held Prize with David Steurer, a professor of theoretical computer science at ETH Zurich, for “revolutionary contributions to the understanding of optimization and complexity in computer science, work that has relevance for solving the most difficult and intractable of computing problems.” The winners will share the $100,000 prize.
Doudna, a Howard Hughes Medical Institute investigator, received the 2018 NAS Award in Chemical Sciences for “pioneering discoveries on how RNA can fold to function in complex ways” and the invention, with Emmanuelle Charpentier, of the CRISPR-Cas9 gene-editing technology.
The winners will be honored in a ceremony on Sunday, April 29, during the National Academy of Sciences’ 155th annual meeting.
Raghavendra’s prize, awarded this year for the first time, was made possible through a bequest from the estate of Michael and Sheila Held. Doudna’s award, established in 1978 and currently supported by the Merck Company Foundation, is accompanied by a medal and a $15,000 prize.
Previous winners of the NAS Award in Chemical Sciences include Paul Alivisatos, a professor of chemistry and UC Berkeley’s executive vice chancellor and provost, chemistry professors emeritus Gabor Somorjai and Robert Bergman, and former chemistry professor Carolyn Bertozzi, who is now at Stanford University.
Another former UC Berkeley faculty member, James Allison, received the NAS’s 2018 Jessie Stevenson Kovalenko Medal “for important medical discoveries related to the body’s immune response to tumors.” He is now at the University of Texas MD Anderson Cancer Center. All are among 18 awards to 21 scientists announced this week.
CRISPR on TED Ideas worth spreading – Ellen Jorgensen, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
CRISPR on TED Ideas worth spreading – Ellen Jorgensen
Reporter: Aviva Lev-Ari, PhD, RN
On same webpage see other CRISPR Talk on TED on the right hand side of the webpage
CRISPR snips a strand of DNA – Visualization of the Process, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
CRISPR snips a strand of DNA – Visualization of the Process
Reporter: Aviva Lev-Ari, PhD, RN
Watch what it actually looks like when CRISPR snips a strand of DNA
Molecular animations are an essential way to demystify and explain complex biological systems. Through the use of stunning imagery and attention to detail, Visual Science and Skoltech have captured the dynamic mechanisms of CRISPR-Cas proteins and their use as research tools.
— Jennifer Doudna, Professor of the Depts. of Molecular and Cell Biology and Chemistry at the UC Berkeley, Executive Director of the Innovative Genomics Institute
You can watch the animation, created by biologists at Russia’s Skoltech Institute and the Visual Science organization, below or at the latter’s website:
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