Fish that adapted to survive in the frigid waters of Antarctica offer a gateway to better understand the genetics of human aging, but the fish are being threatened by climate change, says University of Oregon biologist John Postlethwait.

Thats the message he will deliver Nov. 13 in a Quack Chats pub talk at Eugenes Downtown Athletic Club, 999 Willamette St. The talk will begin at 6 p.m. in the third-floors Ax Billy Grill.

These are remarkably beautiful and complex animals, said Postlethwait, a professor emeritus and member of the UOs Institute of Neuroscience. Climate change is threatening them. We need to study them more before they become extinct or try to change the conditions so that we limit the effects of climate change and maybe reduce the chances of extinction.

Postlethwait traveled five times to Antarctica between 2008 and 2018 with funding from the National Institutes of Health and National Science Foundation. He has studied dragonfish and icefish, primarily the low bone mineralization that evolved in bottom-dwelling icefish to allow them to increase their buoyancy and rise in the water column to feed.

That adaptation of turning off certain genes to become less dense in the absence of swim bladders think lungs in humans began about 30 million years ago as Antarctic waters cooled. Todays seawater temperature there hovers just below the freezing point of fresh water.

The bones in these fish look like old peoples bones, said Postlethwait, who ran a marathon in Antarctica during one of his research trips. As people age, they turn off or turn down the genes that are important for maintaining bone health.

In his talk, Dragons and Icefish: Surviving Frigid Antarctic Seas, hell detail the biology and molecular genetics of these Antarctic fish and how they hold genetic clues that may be useful for understanding anemia, oversized hearts and low bone-mineral density in humans.

The potential human health payoff was boosted earlier this year when an international team that included Postlethwait and six other UO researchers published the complete genome sequence for Antarctic blackfin icefish.

To learn more about upcoming Quack Chats, see the Quack Chats section on Around the O. A general description of Quack Chats and a calendar of additional Quack Chats and associated public events also can be found on the UOs Quack Chats website.

By Jim Barlow, University Communications

Excerpt from:
Pub talk will explore links between icefish and human aging - AroundtheO

A team of UBC researchers used advanced techniques in microscopy and chemical profiling to determine that cannabis flowers with the biggest hairs produce the most CBD, THC, and fragrance-emitting terpenes.

The research was the first to detail the structures and chemical-creating abilities of the three kinds of frostlike pot hairsstalked, sessile, and bulbouscalled glandular trichomes.

Finola, a fast-flowering hemp variety of Cannabis sativa, was used by the team, which included researchers from UBC's Wine Research Centre and the Michael Smith Laboratories.

Teagen Quilichini, the study's co-lead author and a postdoctoral fellow with the botany department, said in an October 28 UBC news release that the study is an important beginning for subsequent research.

Despite its high economic value, our understanding of the biology of the cannabis plant is still in its infancy due to restricted legal access, Quilichinisaid. Trichomes are the biochemical factories of the cannabis plant and this study is the foundation for understanding how they make and store their valuable products.

The paper"Cannabis glandular trichomes alter morphology and metabolite content during flower maturation"appeared in the August 30 edition of the Plant Journal.

Anne Lacey Samuels, a botany professor and principal investigator for the study, said in the release that there could be many downstream benefits of the research.

We found a treasure trove of genes that support the production of cannabinoids and terpenes. With further investigation, this could be used to produce desirable traits like more productive marijuana strains or strains with specific cannabinoid and terpene profiles using molecular genetics and conventional breeding techniques.

The UBC team determined under ultraviolet light that the stalked trichomes glowed blue and had large, distinctive secretory discs of cells that looked like microscopic mushrooms or hamburgers.

We saw that stalked glandular trichomes have expanded 'cellular factories' to make more cannabinoids and fragrant terpenes, co-lead author Sam Livingston said in the release. We also found that they grow from sessile-like precursors and undergo a dramatic shift during development that can be visualized using new microscopy tools."

Livingston, a botany department PhD candidate, noted thatUV lightmight be used todetermine flowers' trichome maturity so growers would know the best times to harvest.

See the original post here:
UBC cannabis researchers find that "frosty" flowers mean potent pot - Straight.com

Graph 1

Vectra Predicts Risk of Radiographic Progression in 1 Year

Myriad Genetics, Inc.

Graph 2

Vectra Predicts Risk of Cardiovascular Events in Patients with RA

Myriad Genetics, Inc.

SALT LAKE CITY, Nov. 09, 2019 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a global leader in precision medicine, announced that its Myriad Autoimmune business unit will present new data on the Vectra test at the 2019 ACR/ARP Annual Meeting being held Nov. 8-13, 2019 in Atlanta, GA. The key findings are that the Vectra test predicts the risk of radiographic progression (RP) within one year, and the Vectra score, in combination with other clinical measures, predicts the risk of a cardiovascular (CV) event in people with rheumatoid arthritis (RA).

A hallmark feature ofrheumatoid arthritisisinflammation, which increases the risk of joint damage, cardiovascular disease and other comorbidities, said Elena Hitraya, M.D., Ph.D., rheumatologist and chief medical officer at Myriad Autoimmune. The data being presented by our academic collaborators at ACR show that the Vectra test accurately measures inflammation and can help predict patients risk of adverse health outcomes, enabling clinicians to tailor precision treatment plans to achieve better outcomes.

Vectra Posters

Title:Predicting Risk of Radiographic Progression for Patients with Rheumatoid Arthritis.Presenter:Jeff Curtis, M.D., M.S., MPH, University of Alabama at Birmingham.Date:Sunday, Nov. 10, 2019. 9:00-11:00 a.m.Location:Poster 466.

This study evaluated the ability of the Vectra test to predict patients individual percentage risk of RP within one year. The analysis included combined data from 973 patients in four cohorts. The results demonstrate that the adjusted Vectra score was a superior predictor of RP within one year compared to DAS28-CRP, CRP, CDAI and swollen joint count. Additionally, the risk of permanent joint damage increased continuously with the adjusted Vectra score, meaning patients with a low adjusted Vectra score had a one to three percent risk of RP in one year, while patients with a moderate-to-high score had between seven and 47 percent risk (Graph 1). Based on these new data, the company is working to enhance the Vectra test report to provide patients with their individual risk of radiographic progression in one year.

To view Graph 1: Vectra Predicts Risk of Radiographic Progression in 1 Year,please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/514919cd-81ca-4084-81df-682fedc1784b

Too often people with RA are over- or under-treated because it is difficult for clinicians to accurately measure inflammation and determine the long-term prognosis of RA patients. As a result, some people are at increased risk of rapid radiographic progression, said Jeff Curtis, M.D., M.S., MPH, lead investigator, rheumatologist and Professor of medicine in the Division of Clinical Immunology and Rheumatology at the University of Alabama at Birmingham. It is critical that clinicians have reliable information when making treatment decisions. Our study demonstrated that the Vectra score was the strongest predictor of radiographic progression, which may help inform treatment plans and prevent future joint damage.

Title:Derivation and Validation of a Biomarker-Based Cardiovascular Risk Prediction Score in Rheumatoid Arthritis.Presenter:Jeff Curtis, M.D., M.S., MPH; University of Alabama at Birmingham.Date:Tuesday, Nov. 12, 2019. 9:00-11:00 a.m.Location:Poster 2350.

This study evaluated 30,751 Medicare patients with RA to develop and validate the Vectra CVD score, which predicts risk for a first cardiovascular (CV) event by combining data from Vectra and clinical measures. The primary CV outcome was a composite of three types of CV events heart attack, stroke, and CV death occurring within 3 years from testing. When the performance of the Vectra CVD score was compared to four other CV prediction models, the Vectra CVD score was a significant predictor of CV risk and was superior to all four other models. Importantly, when risk scores were converted to 3-year percentage risk for having a CV event, approximately 80 percent of patients were found to have a moderate or high risk of a CV event over 3 years, based on risk categories analogous to those of the American College of Cardiology/American Heart Association 2018 guidelines (Graph 2).

To view Graph 2: Vectra Predicts Risk of Cardiovascular Events in Patients with RA, please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/c902b4ec-a3c8-439f-9557-0a9b05631a1f

People with rheumatoid arthritis have almost double the risk of heart attack, stroke and atherosclerosis. Traditional CV risk factors alone do not fully explain the increased rates of CV events in RA, and inflammation is a missing component that is measured by the Vectra test, said Dr. Curtis. In this study, the Vectra CVD score effectively predicted CV risk in people with RA. We believe the Vectra CVD score may assist clinicians to more quickly identify patients at high risk for CV events and target interventions that can be potentially life-saving.

The company plans to publish these new data in peer reviewed medical journal and make the Vectra CVD score available to clinicians in fiscal year 2020. Please visit Myriad Autoimmune at booth #1419 to learn more about Vectra. Follow Myriad on Twitter via @myriadgenetics and follow meeting news by using the hashtag #ACR19.

About VectraVectra is a multi-biomarker molecular blood test that provides an objective and personalized measure of inflammatory disease activity in patients with rheumatoid arthritis. Vectra provides unsurpassed ability to predict radiographic progression and can help guide medical management decisions with the goal of improving patient outcomes. Vectra testing is performed at a state-of-the-art CLIA (Clinical Laboratory Improvement Amendments) facility. Test results are reported to the physician five to seven days from shipping of the specimen. Physicians can receive test results by fax or the private web portal, VectraView. For more information on Vectra, please visit: http://www.vectrascore.com.

About Myriad GeneticsMyriad Genetics Inc. is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, Foresight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the Company presenting new data on the Vectra test at the 2019 ACR Annual Meeting; the Vectra test enabling clinicians to tailor precision treatment plans to achieve better outcomes; the Vectra score helping inform treatment plans and prevent future joint damage; the Vectra CVD score assisting clinicians to more quickly identify patients at high risk for CV events and target interventions that can be potentially life-saving; publishing these new data in peer reviewed medical journal; making the Vectra CVD score available to clinicians in fiscal year 2020; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

Go here to read the rest:
New Studies Demonstrate the Predictive Value of the Vectra Test in People Diagnosed with Rheumatoid Arthritis - GlobeNewswire

Q&A

Penn's Kiran Musunuru talks to us about the technology that has been both praised and criticized for its ability to alter human DNA and potentially cure disease.

Kiran Musunuru is an associate professor of medicine in genetics in the Perelman School of Medicine at the University of Pennsylvania. / Courtesy

CRISPR, the technology being used to edit genes in humans, remains polarizing. On one end, detractors argue that using the technology for certain purposes, like performing gene editing on embryos, is not only dangerous but unethical. On the other end, proponents say CRISPR has the potential to revolutionize human health, and early data shows they might be right. Despite a medical community that is still split on the issue, researchers in the U.S. are kicking tests of the technology into high gear. Several clinical trials have launched in the U.S. testing CRISPRs ability to treat various diseases.

NextHealth PHL spoke with Kiran Musunuru, an associate professor of medicine in genetics at the Perelman School of Medicine at the University of Pennsylvania about the true potential of CRISPR technology and how we can expect it to evolve in the future.

NextHealth PHL: What exactly is CRISPR?Musunru: CRISPR is sort of a catch-all term that covers a variety of technologies. If youre saying CRISPR, youre referring to a broad set of tools that may do it in different ways but are all intended to do a form of gene editing or genome editing.

How do basic CRISPR technologies work?The simplest form of CRISPR, what I call version 1.0, is the original standard CRISPR that most laboratories and companies interested in developing new therapies use. It is a two-component system. There is a protein and an RNA molecule thats about 100 bases in length. The protein and the RNA molecule come together to create what well call a molecular machine and the purpose of this molecular machine is to scan across any DNA molecule it encounters. So if you put the CRISPR-Cas9 into the nucleus of a human cell, this molecular machine will scan the entire genome.

The machine has two key functions built into it; the first is a GPS function. When you change the first 20 bases in a DNA length (the first 20 bases is basically the address) to whatever address you want, the GPS function makes the machine go through the entire genome and find the sequence that matches the address. The second function of this machine is to protect the genome, like a search-and-destroy function. You put in the address, it goes to that matching place in the genome and then it makes a cut in the DNA.

Cutting the DNA is actually a bad thing but the cells have ways to try to fix that break, and the actual editing is a result of the cell trying to fix that break in the DNA, not from CRISPR itself, interestingly enough.

How does CRISPR turn a break in someones DNA into a good thing?There are a few ways this can happen. The safest thing you can do is to break a gene or turn off a gene. The metaphor I like to use is to think of the whole genome as a book, and each chromosome in the genome is a chapter in the book, and each gene is a paragraph in the chapter. Together, it all has a meaning. But lets say you had to turn off a gene, the equivalent of making that break in the DNA would be like tearing the page through that paragraph. So, the simplest thing the cell can do and will try to do is to simply tape that tear back up. But as you can imagine, sometimes you tape it back up and its fine, the paragraph is still legible and the meaning is still there, and it eventually heals and functions like it did before. But in this case, thats actually not what you want. The outcome that you want with CRISPR is that you actually want to turn off the gene, not to rip it and make it the way it was before.

What has to happen is when you make the tear, the tear is so rough, you get those jagged edges and you try to tape it up but it doesnt quite fit, the letters dont quite match up. You tape it up as best as you can but its illegible, some letters are lost, and the meaning of the paragraph is lost. Thats exactly what happens with gene editing, the cell tries to repair that break in the DNA, doesnt get it quite right, and loses some bases and that messes up the gene and turns it off.

However, in this scenario, you cant really control what happens. All you can hope for is that that tear you make is going to mess up the gene and thats okay if all youre trying to do is turn it off. Most of the trials underway now are about turning off the gene, and theyre all taking advantage of the fact that its relatively easy to mess up genes and turn off genes. Just like tearing a page its crude, but its effective.

Theres CRISPR 1.0, this first generation of the technology thats not very precise and is a bit arduous. What are the newest forms of CRISPR and how are they better than earlier versions of the technology? There is a newer form of the technology called base editing that keeps the GPS function intact but removes the cutting function. In place of the cutting function, it attaches another machine onto CRISPR and makes chemical modifications in certain areas. This version of CRISPR is more like a search and replace. CRISPR provides the search but then another machine attached to it is doing the replacing. With base editing you can make more precise changes, but only rarely will it make exactly the type of change you want.

The latest form of CRISPR is called prime editing, and we still dont have a good sense of how well it works because its so new. Whats tantalizing is that it looks like it can turn CRISPR into a precise word processor or an eraser that allows you to erase a letter and put in a new letter. CRISPR is very much a wave of technology, and as it gets better, its going to allow us to do more and more powerful things.

There are some extreme ideas about what CRISPR can do. Some believe scientists can use the technology to alter hair or eye color or give patients superhuman athletic or intellectual abilities. Is any of this possible with CRISPR?It depends on what traits youre talking about changing. Since eye color and hair color are controlled by single genes, you could possibly make a single gene change with CRISPR. The problem is, how do you get CRISPR to go where it needs to go to change your hair or eye color? How do you get it into all your hair follicles or through all the cells in your eye? It might be a simpler change to make, but it might not be easy to do in a live adult. Scientists have now edited human embryos, resulting in live-born people. Theres been a lot of ethical debate about whether thats a good thing. If you want to change something like hair color in a single cell embryo made through in-vitro fertilization, thats a bit different and might not be as difficult.

There are some very complicated things, like intelligence or athletic ability, that are not going to be easy to change. Youd probably have to change hundreds of genes, and thats not going to happen anytime soon. With CRISPR as it is now, maybe you can change one gene; maybe if you really work at it you can change two genes, but hundreds of genes? Youre not going to be able to do that with CRISPR anytime soon.

What has CRISPR been used to treat so far and what could it be used for in the future?There are multiple trials underway to treat rare liver disorders. More recently CRISPR has been used in clinical trials at Penn where at least three patients have been dosed using CAR T immunotherapy. In this case, theyre trying to make patients cells more effective at fighting cancer. But again, that editing is being done outside the body.

There are some things that seem like they would be difficult to treat, but if its the right type of disease and you can get CRISPR to where you need it to go, it might work. One example is in sickle cell disease. The cells that you need to fix in sickle cell disease are in the bone marrow. Fortunately, bone marrow is relatively straight forward to work with. You take the cells out and edit them with some form of CRISPR outside of the body and then put them back in.

Something like cystic fibrosis would be much harder because it affects the entire surface of potentially multiple organs inside the body. Its much harder to deliver CRISPR to all of those places in the body.

There are two other clinical trials that have started in the U.S. One is from a company called CRISPR Therapeutics to treat sickle cell disease and similar blood disorders. Theres another trial underway to treat a genetic form of blindness and this editing would actually happen inside the body.

Originally posted here:
Q&A: Everything You Need to Know About the Future of CRISPR-Cas9 - Philadelphia magazine

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTx), a development-stage biotechnology company leading the field of RNA-editing gene therapy, announces $35.5M in Series A financing, led by New Enterprise Associates (NEA), with additional participation from CureDuchenne Ventures. The new capital will enable the company to extend its growing portfolio of intellectual property, recruit and hire top scientific talent and advance its groundbreaking RNA and protein targeting platforms focused on curing human diseases.

These platforms include the proprietary ShapeTx RNAfix technology that enables direct in vivo targeting and modification of RNA by leveraging proteins such as Adenosine Deaminases Acting on RNA (ADARs), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix platform differentiates from other contemporary genome engineering technologies by engaging natural human cellular machinery to modify RNA.

ShapeTx was founded on the work of Dr. Prashant Mali, Assistant Professor of Bioengineering at UCSD, who during his postdoctoral fellowship in the George Church laboratory at Harvard Medical School pioneered the use of CRISPR in human cells. ShapeTx RNAfix platform is built upon his lab's most recent work demonstrating in vivo use of guide RNAs to recruit native ADARs and to fix mutations in multiple rare genetic disease mouse models.

Our technology can correct mutations or target specific genes in neurodegenerative, oncology, metabolic and rare genetic disorders by hijacking naturally occurring proteins such as ADARs present in our cells using just a short guide RNA. Our proprietary new platform avoids the risk of in vivo immunogenicity and permanent off-target damages commonly associated with CRISPR-based approaches, explained Francois Vigneault, Ph.D., President and CEO, who was previously VP of Research at Juno Therapeutics after a successful co-acquisition of AbVitro, Inc. by Juno and Celgene.

Ed Mathers, Partner at NEA and Board member at ShapeTx, said, One rarely comes across a proprietary technology platform with such transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development. The team has shown us an exciting demonstration of the technology in multiple in vivo models, alongside one of the strongest IP estates we have seen in the field. NEA looks forward to backing the company in future rounds as they move the technology toward the clinic.

While the ShapeTx platform will be enabling for many other genetic diseases, Dr. Malis in vivo proof of concept in Duchenne Muscular Dystrophy was quite exciting and could potentially lead to a cure for families suffering from such a debilitating disorder, said Debra Miller, CEO and Founder of CureDuchenne and CureDuchenne Ventures.

The ShapeTx Series A financing coincides with the formation of a world-class Scientific Advisory Board comprised of foremost global experts in genomics, bioengineering, and gene editing, including George Church Ph.D., James Collins Ph.D., and Don Cleveland Ph.D. The scientific advisory board will serve as strategic advisors and ensure that the research and development of its platforms meet the highest standards of scientific merit.

Prashant and Francois are some of the most innovative and brilliant individuals that have come through my lab over the years, and it will be impressive to see these two disrupt the field of gene therapy with this paradigm-shifting technology, said Dr. George Church, Professor in Genetics at Harvard Medical School and member of the ShapeTx Scientific Advisory board.

Shape Therapeutics Scientific Advisory Board Members:

George Church, Ph.D.

George Church Ph.D., world-famous geneticist, molecular engineer, and chemist. He developed the methods used for the first genome sequence & million-fold cost reductions since, as well as pioneered many of the CRISPR advances in genome editing. He is currently a Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT). He is Director of the U.S. Department of Energy Technology Center and Director of the National Institutes of Health Center of Excellence in Genomic Science. He has received numerous awards, including the 2011 Bower Award and Prize for Achievement in Science from the Franklin Institute and election to the National Academy of Sciences and Engineering.

James Collins, Ph.D.

James Collins Ph.D., is one of the pioneers of the field of synthetic biology and has made multiple synthetic biology and bioengineering breakthroughs in biotechnology and biomedicine. He serves as the Termeer Professor of Medical Engineering & Science and Professor of Biological Engineering at MIT, as well as a member of the Harvard-MIT Health Sciences & Technology Faculty, and core member of the Wyss Institute. His many awards include a Rhodes Scholarship, a MacArthur Genius Award, a National Institutes of Health Directors Pioneer Award. Jim is also an elected member of the National Academy of Sciences, the National Academy of Engineering, the National Academy of Medicine, the American Academy of Arts & Sciences, as well as a charter fellow of the National Academy of Inventors.

Don Cleveland Ph.D.

Don Cleveland Ph.D. is an award-winning inventor and pioneer in the field of Antisense Oligonucleotide (ASO) and their uses in gene therapy. He was recently awarded the Breakthrough Prize in Life Sciences for his work on the pathogenesis of disease and ASO-mediated treatment approaches in ALS and Huntingtons disease. Don is currently Professor of Medicine and Department Chair of Cellular and Molecular Medicine and Neurosciences at the University of California at San Diego, and Head, Laboratory for Cell Biology at the San Diego branch of Ludwig Cancer Research. He has made pioneering discoveries on the mechanisms of chromosome movement and cell-cycle control during normal cellular division, as well as the principles of neuronal cell development and the relationship to defects that contribute to inherited neurodegenerative disease.

About Shape Therapeutics, Inc.

Shape Therapeutics, Inc. is creating the worlds leading RNA and protein targeting platforms focused on the cure of human diseases. These include developing precision RNA editing through proteins such as ADAR (Adenosine Deaminase Acting on RNA), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix technology allows for the editing of RNA using natural human cellular machinery, limiting the risk associated with immunogenicity, cellular toxicity, or off-target DNA editing. The teams founders include Prashant Mali, Ph.D., Francois Vigneault, Ph.D., and John Suliman. ShapeTx is headquartered in Seattle, Washington, with a satellite site opening in Cambridge, Massachusetts. For additional information, visit http://www.ShapeTx.com.

About NEA

New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With more than $20 billion in cumulative committed capital since the firm's founding in 1978, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 225 portfolio company IPOs and more than 375 acquisitions. For additional information, visit http://www.nea.com.

About CureDuchenne Ventures

CureDuchenne Ventures supports Duchenne research by using philanthropic donations to encourage the development of new Duchenne drugs. Through an impact financing model, we can provide equity or royalty financing to biotech and pharmaceutical companies. CureDuchennes portfolio includes 16 wide-ranging projects with several successful exits. Investments from CureDuchenne Ventures have successfully de-risked and leveraged more than $2.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne. For additional information, visit https://www.cureduchenne.org/ventures/.

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Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to...