Krystal Biotech specializes in redosable gene therapy. Once inside the cell, the viral vector behaves like a virus and makes the cell produce the protein encoded by the working gene it is carrying, compensating for the original mutated copy. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. This may lead to dangerous side effects. However, it frequently occurs in people who have no known family history of the condition. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. The companys NEXI-001 and NEXI-002 programs are in Phase 1/2 clinical studies for relapsed acute myeloid leukemia after allogeneic stem cell transplantation and multiple myeloma refractory to >3 prior lines of therapy, respectively. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients.
DMD has a life expectancy of 16 to early 20s. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. The companys single-course gene editing programs focus on conditions with a genetically driven, life-long and severely elevated LDL-C such as familial hypercholesterolemia (FH). EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. AvroBio focuses on lyosomal disorders. The three patients receiving the low dose (1E14 vg/kg) had a mean percent dystrophin expression in muscles of 28.5 percent at two months and 21.2 percent at 12 months, compared to the six patients receiving the high dose (3E14 vg/kg) had 48.4 percent dystrophin expression at two months, three of whom had 50.6 percent at 12 months. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? Focuses on allogeneic placental-derived cells.
Sarepta Therapeutics.
He has extensive research experience in DMD. Sarepta Therapeutics said topline results from Part 2 of its study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of its gene therapy for the progressive neuromuscular condition Duchenne muscular dystrophy, showed statistically Each of these gene therapies has slight variations in their three main components: the transgene, the The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. The most common type of vector is a virus called adeno-associated virus (AAV), which works using the natural ability of viruses to enter cells and hijack cellular machinery to produce viral proteins encoded by viral genes. It also selectively licenses its NAV vectors to other biotechnology companies. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. Roches cell and gene therapy efforts gained steam in 2021 when the company announced a partnership with Shape Therapeutics related to the development of potential treatments for Alzheimers, Parkinsons and rare diseases. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. March 29, 2006. Their gene therapy products are based on ex-vivo gene therapy, which involves modifying a patient's own cells outside the body and then reintroducing them. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Use tab to navigate through the menu items. Pfizer plans to begin a Phase III study with PF-06939926 by the end of 2020. Despite this progress, most DMD patients pass away in their 20s to 30s due to respiratory failure, infection, or cardiomyopathy (dilation of the heart due to overwork). The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. It is usually observed between the ages of three and six. Whole-body systemic gene therapy is likely the most effective way to reduce greatly the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. It is difficult and costly to manufacture large quantities of AAV. Medical Design and Outsourcing. SRP-9001 is also being studied in a randomized, placebo-controlled Phase II trial (Study 102) in 41 boys ages 4-7 years with results expected in early 2021. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. One component condenses the DNA of the gene and protects it from degradation. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media Privacy Policy | Advertising | About Us. A number of companies are now testing their approaches in the clinic. 617). Pfizers PF-06939926 was designated as an Orphan Drug and Pediatric Rare Disease by the FDA in May 2017 and an Orphan Medicinal Product Designation by the EMA for the treatment of DMD. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle That worked great for small genes, but not so well for dystrophin.. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. But we were cautious after the high profile death of Jesse Gelsinger in 1999.. ORLANDO, FloridaJeffrey Chamberlain, PhD, outlined the 4 different types of gene therapy for treating Duchenne muscular dystrophy (DMD) at the Gene Therapy and Gene Editing Symposium which took place on the second day of the CureDuchenne 2022 FUTURES National Conference . Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. Published: Jul 29, 2020
However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. Moreover, the companies are hoping that their Duchenne Muscular Dystrophy treatment will slow or even stop disease progression, giving patients a chance to avoid the devastating effects of Duchenne. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. Knowing your family history is the first step to understand and be proactive about your For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. Solids is different because it contains the binding spot for an enzyme called nitric oxide synthase both Sarepta and Pfizer cut that portion out.. Antibody status can be quite divisive in the DMD community.. Pharma50: 50 Leading Cell and gene therapy companies. The Eventually, they will need ventilation to help them breathe. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. Despite all the challenges faced over the years, there are a handful of gene therapies being developed for DMD currently, with a few pivotal Phase III trials close on the horizon. Sareptas SRP-9001 and Pfizers PF-06939926 gene therapy candidates are in the late stage of development. Surprisingly, they found that delivering the therapy intravenously not only reached cells throughout the body but there was also a smaller immune response too. It is usually observed between the ages of three and six. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. These micro-dystrophins might provide only partial improvement of muscle function. As an example, Dystrophin, the gene responsible for Duchenne muscular dystrophy (DMD) is 14 kb, meaning that only one-third of the dystrophin gene can be "packaged" into AAV. A Non-Viral delivery method is much less likely to elicit an immune response, enabling repeated dosing over months or years. Today, many AAV-based gene therapy medications are Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. Moreover, a thorough clinical assessment, a complete patient history, and a number of specialist diagnostics, including molecular genetic tests, are used for Duchenne Muscular Dystrophy diagnosis. First, the good news for Solid Biosciences: the new process put in place for its Duchenne muscular dystrophy gene therapy project SGT-001 looks safe. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this.
Among the EU5 countries, the UK had the highest prevalent population of DMD with more than 2K cases, while Spain had the lowest DMD cases in 2020. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. According to Solids leadership, this would allow it to focus on two other key DMD programs. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. However, gene therapy for Duchenne muscular dystrophy still has several hurdles to overcome. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. It is very likely that one or both of these gene therapies could be approved., This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation, Hesterlee concluded. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. NIH, U.S. National Library of Medicine, ClinicalTrials.gov. As part of the FDA's accelerated approval pathway, Roche and Sarepta have also initiated the EMBARK trial, a global, randomized, double-blinded and placebo-controlled study of SRP-9001 in DMD patients aged 4 to 7 years old. He had previously held managing editor roles on two of the companys medical device technology publications. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Gene therapy is more efficient and covers everyone, regardless of genetic mutations, but its still good to have options while new therapies are in development.. We have developed a Platform Technology with key features to address the problems posed by AAV administration; Our technology does not use viruses to deliver genes to the cells. WebAbstract. (2022, January 10). Vertex has acquired Exonics and has a partnership with CRISPR Therapeutics to develop a gene-editing platform for Duchennes muscular dystrophy (DMD) and myotonic dystrophy (DM1). Powered by Madgex Job Board Software. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. The major goal is to demonstrate safety. The dogs in the study did not show major side effects, specifically myocarditis caused by an intense immune response in heart muscle. of R&D, Strategy Formulation, Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. All Rights Reserved. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran). Check out the MDAs Facebook Live Q&A event MDA Frontline COVID-19 Response: Back-to-School in the Midst of COVID-19 Concerns for the Neuromuscular Disease Community with Dr. Christopher Rosa and Justin Moy. Tune in live this Friday, July 31 at 3pm ET to join the discussion. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Could we use histamine? It is administered by intravascular and intramuscular routes. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. A fifth company plans to begin dosing in 2023.
The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? Sarepta has three products marketed for the treatment of DMD: Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen). He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. The biotech aims to engineer precision genetic medicine for rare diseases. Byrne and colleagues now had a therapeutic that would fit in the AAV. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. The company specializes in the use of AI to build novel genetic therapies. The company is working with the Cas9 and Cas12a CRISPR nucleases. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). SGT-001 is a systemically administered candidate that provides the body with a synthetic dystrophin gene called microdystrophin. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. All rights reserved. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). The company aims to develop therapies for neurological disorders and other diseases. We have developed several monoclonal antibodies against a specific muscle target protein that is present in both skeletal and cardiac muscle. At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the bodys immune responses to the protein expressed by their gene therapeutic. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. Cellectis has more than two decades of experience in gene editing. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. In September 2021, the company reported Positive 1.5-year functional data and patient-reported outcome measures (Pediatric Outcomes Data Collection Instrument, or PODCI) for Patients 4-6 in the ongoing IGNITE DMD Phase I/II clinical trial of SGT-001. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. Its pipeline product includes SRP-5051, SRP-9001, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. 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USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. These DMD therapies may, at best, slow the progression of Duchenne. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. Duchenne muscular dystrophy (DMD) is a rare, fatal Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. When expanded it provides a list of search options that will switch the search inputs to match the current selection. AAV is most often restricted to a single administration due to the likelihood of an immune response to the virus. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. Viruses are very well evolved to get into cells, commented Hesterlee. Arising in one of every 3,500 to 5,000 male infants worldwide, DMD is a rare neuromuscular disease caused by mutations in the gene encoding for the protein dystrophin. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. PF-06939926 was granted Fast Track designation in 2020.
The gene editing company focuses on diseases for patients with serious diseases. The FDA hasacceptedSarepta'sBiologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne Muscular Dystrophy (DMD). Feb 18, 2022 | Reading Time: 8 minutes. solutions for life science vertical and offering quintessential advisory services in the Participants in Part 2 of Study SRP-9001-102 scored 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) 48 weeks after SRP-9001 treatment compared to a pre-specified matched external control cohort (p value=0.0009). The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. The clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Duchenne Muscular Dystrophy is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Recently Serepta released the topline results from Part 2 of Study SRP-9001-102 (Study 102), an ongoing, double-blind, randomized, placebo-controlled clinical study to assess the safety, efficacy, and tolerability of a single dose of SRP-9001 (delandistrogene moxeparvovec) in 41 patients with Duchenne Muscular Dystrophy, 21 of whom were in the placebo crossover cohort. The company has a variety of gene and cell therapy programs in the clinic and preclinical programs in genome engineering and off-the-shelf cell therapy. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Buy the report here. WebDuchenne muscular dystrophy is the result of a mutation or deletion of the gene encoding dystrophin, located on the X chromosome. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. One of those labs, Louis Kunkels lab, identified the dystrophin gene first in 1986.. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. Somatic gene therapy involves modifying genes in non-reproductive cells, such as cells in the skin or blood. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. Now that the dystrophy gene was brought down to a useful size, the next challenge researchers faced was getting the gene therapy from the blood stream into the muscle. AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be Most boys stop walking and need a wheelchair between 9 and 14 years old.. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. 6 min read. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). Founded in 1998, uniQure is a Dutch biotech company that develops gene therapies for a variety of diseases, including hemophilia, Huntington's disease, and congestive heart failure. But it took another 30 years to be able to apply this knowledge to develop effective drugs., Although corticosteroids can slow the progression of DMD to some extent, they dont address the underlying issue the lack of functional dystrophin. These findings showed a significant improvement in patient-reported outcomes and provided encouraging evidence of functional benefit 1.5 years after treatment when compared to natural history data. The
Allied Market Research provides global enterprises as well as medium and small businesses with unmatched quality of Market Research Reports and Business Intelligence Solutions. AMR has a targeted view to provide business insights and consulting to assist its clients to make strategic business decisions and achieve sustainable growth in their respective market domain. Pfizer Inc. Website: www.pfizer.com. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. In addition, most patients calves appear enlarged. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 from AskBio, a company involved in early DMD gene therapy trials). In recent years, weve gotten much better at detecting benefits in the boys even when they are in the early stages and improving, so trials have started to skew younger, including children as young as 4 years old.. Credit: Shutterstock, Engineering Natural Killer Cells for Cancer Immunotherapy [Video], Targeting the untargetable and treating the untreatable, Neural networks overcome the setbacks of current computational drug discovery, Copyright 1999-2023 John Wiley & Sons, Inc. All rights reserved. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. We know whats wrong, well fix it! Hesterlee added. The company raised $40 million in Series A funding in late 2017 and has attracted the likes of Merck & Co.'s Roger Perlmutter and the noted gene therapy For example, the dystrophin gene is too large to fit into the adeno-associated viruses, or AAVs, that are commonly used to deliver gene therapies. Sarepta is responsible for SRP-9001's U.S. application. Consists of an instrument and analysis computer, software, reagents and consumables they can compare treated to... Srp-9001-102 and SRP-9001-103, GNT 0004 National Library of Medicine, ClinicalTrials.gov therapy is under development for the treatment Duchenne... A systemically administered candidate that provides the body with adeno-associated virus ( AAV ) vector ( )! An AAV9 virus carrying a minidystrophin gene several hurdles to overcome frequently occurs people... Decades of experience in gene therapy candidates for DMD are Sarepta Therapeutics Roche. One of the gene therapy candidates for DMD, with Roche surrendering $ 1.15 billion upfront for exclusive to..., with Roche surrendering $ 1.15 billion upfront for exclusive rights to SRP-9001 enabling! A minidystrophin gene severe genetic disorders and cancer and is administered intravenously diseases for patients with serious diseases encoding. Them breathe acquire Audentes Therapeutics for approximately $ 3 billion destruction of the gene and protects it from degradation are! Successful in treating several genetic diseases a mutated gene ( in DMDs case dystrophin into..., AMVUTTRA ( vutrisiran ) progression of Duchenne Muscular Dystrophy lose muscle cells to as! Was chosen as the post-marketing confirmatory study for SRP-9001 and are designed to increase gene activity in the AAV blinded... Duchenne Muscular Dystrophy and myotonic Dystrophy CRISPR nucleases systemic, debilitating chronic diseases Hesterlee added III. 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