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Category Archives: Gene Medicine

The science behind those afternoon naps Harvard Gazette – Harvard Gazette

How often a person takes daytime naps, if at all, is partly regulated by their genes, according to new research led by investigators at Harvard-affiliated Massachusetts General Hospital (MGH) and published inNature Communications.

In this study, the largest of its kind ever conducted, the MGH team collaborated with colleagues at the University of Murcia in Spain and several other institutions to identify dozens of gene regions that govern the tendency to take naps during the day. They also uncovered preliminary evidence linking napping habits to cardiometabolic health.

Napping is somewhat controversial, says Hassan Saeed Dashti of the MGH Center for Genomic Medicine, co-lead author of the report with Iyas Daghlas, a medical student at Harvard Medical School (HMS). Dashti notes that some countries where daytime naps have long been part of the culture (such as Spain) now discourage the habit. Meanwhile, some companies in the United States now promote napping as a way to boost productivity. It was important to try to disentangle the biological pathways that contribute to why we nap, says Dashti.

Previously, co-senior author Richa Saxena, principal investigator at the Saxena Lab at MGH, and her colleagues used massive databases of genetic and lifestyle information to study other aspects of sleep. Notably, the team has identified genes associated with sleep duration, insomnia, and the tendency to be an early riser or night owl. To gain a better understanding of the genetics of napping, Saxenas team and co-senior author Marta Garaulet of the department of physiology at the University of Murcia, performed a genome-wide association study (GWAS), which involves rapid scanning of complete sets of DNA, or genomes, of a large number of people. The goal of a GWAS is to identify genetic variations that are associated with a specific disease or, in this case, habit.

For this study, the MGH researchers and their colleagues used data from the UK Biobank, which includes genetic information from 452,633 people. All participants were asked whether they nap during the day never/rarely, sometimes or usually. The GWAS identified 123 regions in the human genome that are associated with daytime napping. A subset of participants wore activity monitors called accelerometers, which provide data about daytime sedentary behavior, which can be an indicator of napping. This objective data indicated that the self-reports about napping were accurate. That gave an extra layer of confidence that what we found is real and not an artifact, says Dashti.

Several other features of the study bolster its results. For example, the researchers independently replicated their findings in an analysis of the genomes of 541,333 people collected by 23andMe, the consumer genetic-testing company. Also, a significant number of the genes near or at regions identified by the GWAS are already known to play a role in sleep. One example isKSR2, a gene that the MGH team and collaborators had previously found plays a role in sleep regulation.

Digging deeper into the data, the team identified at least three potential mechanisms that promote napping:

This tells us that daytime napping is biologically driven and not just an environmental or behavioral choice, says Dashti.

Some of these subtypes were linked to cardiometabolic health concerns, such as large waist circumference and elevated blood pressure, though more research on those associations is needed.

Future work may help to develop personalized recommendations for siesta, says Garaulet.

Furthermore, several gene variants linked to napping were already associated with signaling by a neuropeptide called orexin, which plays a role in wakefulness. This pathway is known to be involved in rare sleep disorders like narcolepsy, but our findings show that smaller perturbations in the pathway can explain why some people nap more than others, says Daghlas.

Saxena is the Phyllis and Jerome Lyle Rappaport MGH Research Scholar at the Center for Genomic Medicine and an associate professor of anesthesia at HMS.

The work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Heart, Lung, and Blood Institute, MGH Research Scholar Fund, Spanish Government of Investigation, Development and Innovation, the Autonomous Community of the Region of Murcia through the Seneca Foundation, Academy of Finland, Instrumentarium Science Foundation, Yrj Jahnsson Foundation, and Medical Research Council.

The science behind those afternoon naps Harvard Gazette - Harvard Gazette

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RARE-X Announces the Expansion of its Board of Directors to Support the Organization’s Growth and Launch Efforts – WFMZ Allentown

ALISO VIEJO, Calif., Feb. 10, 2021 /PRNewswire-PRWeb/ -- RARE-X today announced three new board members who will help support the nonprofit's work in structured patient data collection, responsible data sharing, and the promise of its Federated Data Sharing Platform for data sharing and analysis. The new board members are Cynthia Grossman, PhD, director at Biogen; Jason Colquitt, CEO of Across Healthcare; and Simon Frost, CEO of Tiber Capital Group.

"The additions of Cynthia Grossman, PhD, Jason Colquitt, and Simon Frost to the board are very strategic. All bring a depth of knowledge in patient advocacy, health tech, scaling-up organizations, and operational excellence," said Nicole Boice, RARE-X Co-Founder/Executive Director. "We are honored to have them join an already extraordinary board and thrilled to channel their expertise, talent, and energy into helping RARE-X build towards the future."

Cynthia Grossman, PhD, is a director at Biogen, leading the MS PATHS program, a collaborative research network aimed at generating evidence to improve outcomes for patients living with Multiple Sclerosis. Prior to joining Biogen, Cynthia was director at FasterCures, a center of the Milken Institute. Before joining FasterCures, she was chief of the HIV Care Engagement and Secondary Prevention Program in the Division of AIDS Research (DAR) at the National Institute of Mental Health (NIMH). Cynthia has spent her career working to improve health by expanding opportunities for patients' perspectives to shape the processes by which new therapies are discovered, developed, and delivered. Cynthia graduated Phi Beta Kappa from Earlham College with a B.A. in psychology and biology and earned her Ph.D. in clinical psychology from the University of Vermont. She has been the recipient of a National Science Foundation Incentives for Excellence Scholarship, an NIH Ruth L. Kirschstein National Research Services Award, and a Postdoctoral Fellowship in Pediatric Psychology at the Warren Alpert Medical School of Brown University.

Jason Colquitt is CEO of Across Healthcare, a company he founded in 2012, leveraging his 20+ years in the healthcare technology field. His work has caused positive disruption within the healthcare industry as he has partnered with many organizations ranging from small start-ups to some of the world's largest health companies including Greenway Health, Walgreens Boots Alliance, Quintiles, IQVIA, Cystic Fibrosis Foundation, Muscular Dystrophy Association, American College of Surgeons, and American Heart Association. Jason has worked directly with patients, caregivers, physicians, regulators, and researchers. Jason was diagnosed with Carnitine Palmitoyltransferase II Deficiency (CPT II), a rare mitochondrial disease. He has used his experiences and technical background to help the rare disease community. Jason holds a Bachelor's degree in Applied Mathematics from Auburn University.

Simon Frost is the CEO of Tiber Capital Group. Before joining Tiber Capital Group, he was the chief investment officer of Greencourt Capital, a public company with approximately $1 billion in real estate assets. Before joining Greencourt Capital, Simon was president and COO of Key Properties. He was also the co-founder of The American Home, one of the largest single-family rental aggregators in the United States. Simon holds Bachelor's and Master's degrees in economics from Cambridge University in England, and a Bachelor's degree in finance from the University of South Africa. Simon serves as director of both Cure AHC and Hope For Annabel, charities dedicated to finding therapies for Alternating Hemiplegia of Childhood.

The current RARE-X Board of Directors includes: Betsy Bogard, head of program and alliance management within the 4:59 Initiative at 5AM Ventures; Nicole Boice, co-founder and executive director of RARE-X; Jason Colquitt, CEO of Across Healthcare; Wendy Erler, vice president of Patient Experience, STAR and Advocacy at Alexion Pharmaceuticals; Simon Frost, CEO of Tiber Capital Group; Peter Goodhand, CEO of Global Alliance for Genomics and Health; Cynthia Grossman, PhD, director at Biogen; Walt Kowtoniuk, PhD, COO of MOMA Therapeutics and venture partner at Third Rock Ventures; Craig Martin, president of Rithm Health and interim CEO at Global Genes; Katherine Maynard, principal at PWR; Angeli Moeller, PhD, head of Pharma Informatics International at Roche; David Pearce, PhD, president of Innovation and Research for Sanford Health; Anthony Philippakis, MD, PhD, chief data officer at Broad Institute; John Reynders, PhD, chief data scientist at Reynders Consulting; Morrie Ruffin, co-founder and board member of ARM Foundation for Cell and Gene Medicine and managing partner, Adjuvant Partners; Alvin Shih, MD, president and CEO at Catamaran Bio.


RARE-X is a 501(c)(3) patient advocacy organization focused on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by rare disease. Enabled by best-in-class technology, patients, researchers, and other technology vendors, RARE-X will gather structured, fit-for-purpose data to share broadly, benefitting from 21st-century governance, consent, and federated data sharing technology. RARE-X is building the largest collaborative patient-driven, open-data access project for rare diseases globally. For more information, visit

Media Contact:

Tom Hume, Marketing Communications RARE-X

Media Contact

Tom Hume, RARE-X, 7602144863,


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RARE-X Announces the Expansion of its Board of Directors to Support the Organization's Growth and Launch Efforts - WFMZ Allentown

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Pfizer to nearly halve COVID-19 vaccine production timeline, sterile injectables VP says – FiercePharma

With an upsized production goal of 2 billion COVID-19 vaccine doses this year, Pfizer and its German partner BioNTech arent resting on their laurels now that their shot, Comirnaty, has emergency nods in the U.S., Europe and beyond. As the companies continueto build out capacity, manufacturing efficiency is getting its own boost, Pfizerrevealed.

The time it takes the companyto produce a COVID-19 vaccine batch could soon be cut from 110 days to an average of just 60, Chaz Calitri, vice president of sterile injectables, told USA Today. We call this Project Light Speed, and its called that for a reason, he said. Just in the last month, weve doubled output.

One element teed up for acceleration is DNA productionthe first step inPfizers vaccine manufacturing process, Calitri explained. Making that DNA originally took 16 days, but the process will soon take just nine or 10 days, he said.

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RELATED:Pfizer, Johnson & Johnson balk at shareholders' push for COVID-19 vaccine pricing info

Production efficiencies aside, the company is also looking to dial up capacity with the addition of new manufacturing lines atall three of its U.S. plants, USA Today said.Demand for a functional shot meant Pfizer didnt have the span of several years typically required to refineits manufacturing process, so the company is improving as it goes, Calitri noted.We just went straight into commercial production," he said.

Engineers took an eye to improving manufacturing the moment vials started coming off production lines, which led the company to make a lot of really slick enhancements, he added.

A Pfizer spokesperson confirmed Calitris comments to Fierce Pharma via email.

RELATED:First-to-market Pfizer expects a whopping $15B from its COVID-19 shot in 2021

Pfizer and BioNTechs manufacturing network depends on six facilities split between Europe and the U.S. Stateside, the vaccine starts its life at Pfizers Chesterfield, Missouri, plant, where the DNA is produced. It then heads to the companys facility in Andover, Massachusetts, for transcription into mRNA, before finally making its way to Kalamazoo, Michigan for fill-finishwith lipid and lipid nanoparticle production and formulation taking place somewhere prior to that final step.Calitri heads up operations at the Kalamazoo plant.

Pfizer and BioNTechs mRNA-based vaccine last year became the first COVID-19 shot authorized in Europe and the U.S. On deck to supply hundreds of millions of doses to those two regions alone, BioNTechs CEO Uur ahin recently said the companies would boost their 2021 output target to 2 billion doses from a prior goal of 1.3 billion.

At the time, ahin pinned those production hopes on six global manufacturing sites tapped in the companies alliance, including a facility in Marburg, Germany, that he said was expected to go live by the end of February.

RELATED:Could combining Pfizer's and AZ's COVID-19 vaccines fill supply gaps? U.K. researchers aim to find out

A little more than a week later, the biotech won approval to start manufacturing itsvaccine at the Marburg site, which employs 300 people and is set to produce up to 750 million doses annually, German news outlet Hessenschau reported.

The announcement ran up against news that BioNTech was carrying out a factory upgrade in Puurs, Belgium that would allow itto deliver significantly more doses in the second quarterthough that production boost came with a catch: namely, a short-term disruption of supply in Europe, Canada and a few other countries.

Meanwhile, in a sign of the unconventional alliancesCOVID-19 has fostered, Pfizer and BioNTech recently got some added manufacturing muscle from two Big Pharma rivals. Sanofi in late January said it would produce more than 100 million Comirnaty doses in Europe in 2021, with the first deliveries from its site in Frankfurt, Germany, expected by August, a company spokesperson told Fierce Pharma.

Just a few days later, Swiss drugmaker Novartis said it would pitch in, too, agreeing to carry out fill-finish work at its facility in Stein, Switzerland, where production is pegged to start in the second quarter.

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Cancer gene sequencing and an unexpected transmission – Health Report – ABC News

The New England Journal of Medicine has a potentially disturbing report from Japan of two little boys, one aged two, the other aged six, diagnosed with lung cancer (they are not related) whose mothers had cervical cancer. Genome sequencing found that the tumours in the kids, although they seemed very different from the mothers, were genetically related to their mum's tumour. The assumption was that cancer cells had spread to the boys in the physical process of being born.

Dr Alison Brand is director of gynaecological oncology at Westmead Hospital in Sydney. Welcome to the Health Report, Alison.

Alison Brand: Thanks for having me.

Norman Swan: So have you ever heard of this before?

Alison Brand: No, I had not heard of this before this and, I have to say, it's virtually unheard of, and the New England Journal of Medicine, which is the peak journal in medicine to publish a case report. So you know if they've published this case report they have really examined the data very closely to verify that it is true. And I think we have to do believe some of it because it has been published in the New England Journal of Medicine.

Norman Swan: Let me just divert from the core story here, which is about cervical cancer moving to the kids and getting into their lungs. You're a gynaecological surgeon, you do a lot of surgery, what's the story with gynaecological seeding and surgery? There has been a lot of discussion about that, whether in fact you can spread cancer with surgery itself.

Alison Brand: The old wives' tale was that once you open up the belly and let the air in, then the cancer just spreads terribly, and of course we know that's not true. That really came from the fact that when patients had operations many years ago, they found cancer but then they couldn't do much about it, there was no chemotherapy or there was no radiotherapy. So really this whole business of surgery spreading cancer is probably not true at all. We do know that cancer from the mum can sometimes cross the placenta and get into babies, but that's usually haematologic malignancies.

Norman Swan: So it's leukaemia.

Alison Brand: Leukaemia, that's right, and otherand basically the babies then have widespread disease because it got into their bloodstream and then went throughout the body. These particular two case reports are really unusual because it doesn't look like it came transplacentally or through the placenta, it looks like it came as the baby has passed through the birth canal, and landed in the lung, which is the kind of closest place that the babies could breathe in some of the cells that were in the vagina as they pass through the birth canal.

Norman Swan: And because of that you wouldn't think it was human papilloma virus related, which is the cause of cervical cancer, because it was the actual tumour itself that got transmitted.

Alison Brand: Yes, that's right, although we haven't often thought that cancer is catching. I think that's the one thing that we've said; you can't touch someone who has cancer and then catch it, and in many ways you catch lots of viruses, and women can pass their HPV infections in some rare cases to their babies. And so this is unusual in that the cancer has really been caught from the mother and that's highly unusual.

I guess when we look at this we have to look atas we examine any reports, we have to say is this biologically plausible, and I guess in rare cases it is biologically plausible, although you mostly expect that the tumour cells on the top of tumours are really those ones that are often non-viable or not living and therefore can't attach to something and grow there. But I think that the next generation sequencing that they have done here really suggests to us that maybe there is some truth to all of this, albeit rare, rare, rare.

Norman Swan: Is it routine to screen for cervical cancer in pregnancy?

Alison Brand: It is routine that patients should have had a recent screen prior to their pregnancy, and if they haven't, to have one done during pregnancy. What you have to remember though, Norman, is certainly the mother of the first patient had had a normal cervical screen seven months prior to delivering her baby, and it's important because she had a very rare neuroendocrine tumour, so a very rare type of cervical tumour that probably wouldn't have been picked up by screening anyway. But those are very rare tumours, and the vast majority of cervical cancers can be picked up by screening, and certainly are much better picked up by the new screening test that we have that looks at HPV presence.

Norman Swan: And before we go, just tell usbecause the screening program has changed, it now happens every five years if I remember rightly, and you are checking for HPV. So, just give us a very brief outline of the screening program now.

Alison Brand: So it used to be that we looked at the cells on the cervix to check to see whether or not they had precancerous changes. That had up to a 30% false negative rate, and therefore we had to screen more often to make sure that we didn't miss anything. Now we check by looking at what we call high risk HPV virus, which is human papilloma virus, which is known to cause cervical cancer, and we check for that high risk HPV, and because the test is so sensitive, then if there is a negative test, we only need to do the test every five years. And I think the take-home message here for women who are pregnant is that we shouldn't worry so much about giving your baby cancer from you, what we should really worry about is making sure that we prevent cancer in the first place by having regular screening and, if eligible, making sure your boys and girls have vaccinations.

Norman Swan: Alison, thank you for joining us.

Alison Brand: Thank you.

Norman Swan: Dr Alison Brand is director of gynaecological oncology at Westmead Hospital in Sydney.

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Cell and Gene Therapies Shatter Prior Records, with Continued Growth Expected – BioSpace

Cell and gene therapies achieved record growth in 2020, surpassing prior high points in terms of financings and approvals. Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), painted a rosy picture despite a few challenges at the 2021 Cell & Gene State of the Industry Briefing during this years Biotech Showcase, held virtually January 11-15.

2020 was a record-shattering year for financing, with $19.9 billion raised in 2020, up from the $9.8 billion raised in 2019 and the $13.3 billion raised in 2018 the previous record, she said.

Follow-on financings, at $6.8 billion, accounted for the largest portion of funding, followed by venture capital at $5.6 billion and IPOs at $3.7 billion. Upfront payments from partnerships totaled $3 billion and private placements totaled $1.2 billion.

The number of large financings exceeding $200 million grew noticeably. Sana Bios $700 million private financing, announced last June, topped the field. Iovance and bluebird bio gained the most from follow-on financing, with fundings of $604 million and $575 million, respectively. Legend Bio led the list of IPOs, raising $487 million last May.

Large pharma continued to buy-in to biotechs for research, development, commercialization, and licensing agreements focuses largely on oncology and CNS disorders, Lambert said. She cited Sangamo, with two major collaborations with Biogen ($350 million) and Novartis ($75 million), though adding, Bayer is especially active.

In the stock market, Regenerative medicine companies outperformed the NASDAQ Biotech Index, she continued. Although stock prices for virtually everything plummeted in mid-March, biotech stocks rebounded. Cell based immuno-oncology (IO) share prices increased 80% from their January 1 levels. Gene therapy was close behind, with a 70% increase, and all publicly traded regenerative medicine companies saw a 50% increase.

Given the overall environment, it seems safe to predict that 2021 will be another good year for regenerative therapies.

Currently, there are approximately 1,100 gene, cell and tissue-based therapeutic developers throughout the world. Of those, the overwhelming majority are in North America, with 543. We saw a lot of growth in China, Japan, and Korea, she said, with 295 companies. Europe boasts 209.

Globally, 1,220 clinical trials are underway for regenerative medicine.

More than 100 clinical trials commended in the fourth quarter alone, Lambert said. Roughly, they are divided evenly among cell, gene, and IO. With 152 trials in phase III and nearly half of those in gene therapy, this offers strong support for predictions by both the FDA and EMA that there will be 10 to 20 advanced therapies approved each year through 2025.

What people often forget, Lambert said, quoting Amy Price, a mother of two gene therapy recipients, is that cell and gene therapies arent some fantastical futuristic thing. Two of the Price children received gene therapy in a clinical trial 10 years ago for metachromatic leukodystrophy (MLD), a historically fatal disease. That drug, Libmeldy, by Orchard Therapeutics, was approved in Europe in 2020, making it one of the most significant milestones of the year.

The benefits of cell and gene therapy have expanded beyond experimental treatments.

Patients are continuing to benefit from innovative therapies, Lambert said. We saw a significant number of gene therapy approvals in 2020. In addition to Libmeldy, she cited approvals of Zolgensma (by Novartis Gene Therapies) in Europe, Japan, and Canada; Tecartus (by Kite, a Gilead company) in the U.S.; and Luxturna (by Spark/Roche) in Canada.

Looking forward, oncology, and particularly IO, dominates the regenerative medicine landscape. Some 554 oncology trials are underway.

Investors have invested heavily in this space for some time, and IO comprises 50% of Phase I trials in cell and gene therapy, Lambert noted. Focus is increasing on allogeneic therapies as well as gene editing.

Central nervous system therapeutics are the second most popular therapeutic indication for regenerative therapies for the second year in a row, with 94 trials. 2020 saw promising data from the first-ever attempt iPSC therapy for Parkinsons disease.

Gene editing continues to advance in the clinic, she added. For the first time, a patient was treated with CRISPR therapy in vivo and, later, systemically with CRISPR. CRISPR Therapeutics and Vertex Pharmaceuticals shared data (during J.P. Morgan week) from a sickle cell trial of 20 patients.

Despite these scientific advances and investor enthusiasm, cell and gene therapies face challenges in terms of dosing and delivery, and chemistry, manufacturing and controls (CMC), Lambert admitted. Gene therapy represents almost half of the Phase III pipeline, so we expect to learn a lot quite soon.

While it goes without saying that 2020 was a challenging year, many of the repercussions of the COVID-19-related disruptions remain to be seen. They extend not only to operational and clinical disruptions but also to regulatory backlogs and the politicization of diagnostics and therapeutics, all set against the usual challenges of fast-moving science.

None-the-less, Lambert pointed out, There were some positive developments.

As she said, Medicare approved a new diagnosis related group (DRG) for CAR T cell therapy and promulgated a new rule for outcomes-based therapies, thus enabling a new payment model that ARM deems essential for cell and gene therapies. In Europe, the European Commissions pharmaceutical strategy now recognizes the importance of cell and gene therapy. We are encouraged that we can build on that starting point with the Commission, Lambert said. ARM also is expecting progress on n-of-one therapies for ultra-orphan indications this year.

Looking ahead to 2021, Lambert identified six regenerative therapies on the FDAs docket from Mallinckrodt, bluebird, BMS, PTC Bio, and Gensight Bio. All indications are that 2021 will be a fantastic year of scientific, technological, and clinical progress in this sector, Lambert predicted.

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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics – GlobeNewswire

-- Alliance will assess the use of Sareptas proprietary gene editing technology and Genevants proprietary LNP delivery platform for multiple neuromuscular targets --

-- Sarepta to have options for an exclusive license to Genevants LNP technology for four neuromuscular indications --

-- Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future milestones and royalties --

CAMBRIDGE, Mass., VANCOUVER, British Columbia, and BASEL, Switzerland, Jan. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industrys most robust and expansive lipid nanoparticle (LNP) patent estate,today announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sareptas pipeline for neuromuscular diseases. LNPs offer the potential for a non-viral approach to gene editing and can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues.

Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations - including rare neuromuscular diseases - by permanently altering genes that lead to disease. Sarepta is pursuing a variety of approaches to genetic medicine including exon skipping, gene therapies and gene editing in pursuit of cures for rare diseases.

Under the terms of the agreement, Genevant will design and collaborate with Sarepta in the development of muscle targeted LNPs to be applied to gene editing targets in early stage development. Sarepta will have rights to an exclusive license to Genevants LNP technology for up to four neuromuscular indications, including Duchenne muscular dystrophy. Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from the mid-single to low-double digits on future product sales.

As Sarepta works to advance precision genetic medicine across multiple modalities, weve invested in partnering and research efforts focused on improving the utility and benefit of gene-based medicines and providing the greatest possible outcome to patients. This includes advancing our pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.Genevants established leadership and proven LNP technology offers the potential to deliver gene editing machinery to targeted tissue through a non-viral delivery approach. Applying this science to neuromuscular diseases fits squarely within our mission to translate scientific breakthroughs into meaningful advances for patients whose lives have been impacted by rare disease.

Genevant scientists have been at the forefront of LNP delivery of nucleic acids for over 20 years. Our platform is the most clinically validated in the space and is the delivery technology behind the first nucleic acid-LNP product to have achieved FDA approval, said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation. Efficient, optimized delivery is often the difference between successful and unsuccessful nucleic acid drug development, and we are excited to bring our experience to Sareptas gene editing programs in neuromuscular disease where new options and new approaches are desperately needed.

About Genevant SciencesGenevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Companys scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Companys LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. Genevant Sciences is committed to transforming the future of human health. For more information, please

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please or follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties obligations and responsibilities under the agreement, potential payments and fees and Sareptas right to an exclusive license to Genevants LNP technology for up to four neuromuscular indications; the potential benefits of LNPs, including offering a non-viral approach to gene editing that can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues; the potential for gene editing to revolutionize the treatment of diseases caused by genetic mutations including rare neuromuscular diseases by permanently altering genes that lead to disease; the goal of Genevant to design and collaborate with Sarepta in the development of muscle-targeted LNPs that can be applied to gene editing targets in early stage development; and Sareptas goal to advance its pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Genevant may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in the discovery of any new therapeutic compounds or any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information by Sarepta

We routinely post information that may be important to investors in the 'For Investors' section of our website We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.


Sarepta Therapeutics, Inc.Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566,

Genevant SciencesPete Zorn,

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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics - GlobeNewswire

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