dmd gene therapy companies

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. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. RGX-202 is intended to deliver a transgene encoding a novel microdystrophin with functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). 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. [This feature is a part of 2022s Pharma 50 series.]. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. In fact, the FDA recently granted SRP-9001 Fast Track designation. According to Kornegay, We showed remarkable decline in loss of respiratory function.. According to the market research firm Emergent Research, international cell and gene therapy companies could generate $6.6 billion in revenue by 2027, with a projected CAGR of 19.8% from 2020 to 2027. They also have 12 other exon skipping-based genetic medicines in their pipeline. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. Gene therapy; Cell Get industry leading news, data and analysis delivered to your inbox. Currently, Matthew is a Venture Partner at Medicxi. The companys Tapestri platform can simultaneously. The They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. 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. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). Knowing your family history is the first step to understand and be proactive about your 617). A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. DMD has a life expectancy of 16 to early 20s. 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. Sarepta is headquartered in Cambridge, Massachusetts, the US. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. Although we now know DMD is a genetic disease, it wasnt that long ago that researchers didnt know why or how the disease came about. Today, many AAV-based gene therapy medications are Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. Data are expected to start rolling in late next year. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. 1985 - 2023 BioSpace.com. Corticosteroids help dampen down inflammation, said Hesterlee. 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). He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. They are currently developing gene therapies using CRISPR/Cas9 technology. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. Jeff is an internationally recognized leader in the gene therapy and muscular dystrophy fields and has been a pioneer in AAV micro-Dystrophin gene therapy research and clinical development for DMD. The NAV AAV8 vector, which has been used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12) are used in RGX-202 to support the delivery and targeted expression of genes throughout skeletal and heart muscle. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. Sarepta is responsible for SRP-9001's U.S. application. But the disease doesnt just affect their legs it affects muscles all over their body. Their gene therapy product, SB-525, is currently in clinical trials for the treatment of hemophilia A. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. The boys motor functions rapidly deteriorate with disease progression and, by only 10 to 15 years of age, many boys with DMD are wheelchair-bound. 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. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Gene therapy is an umbrella term for a range of therapies that target the genetic underpinnings of disease. That worked great for small genes, but not so well for dystrophin.. AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. In addition, RNA has potential in treating brain cancer, asMass General Brighamobserves in its list of one dozen potentially disruptive cell and gene therapy innovations. You can compare outcomes to natural disease due to a rich natural history of DMD. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. 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.. In preclinical studies, the presence of the CT domain was shown to recruit several key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle damage in dystrophic mice. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. The company sells a variety of instruments and consumables. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. Before coming to WTWH, he served as content director focused on connected devices at Informa. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. While Solid Biosciences SGT-001 and Regenxbios RGX-202 are in the early stage of development for DMD treatment. 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. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. https://www.alliedmarketresearch.com/request-sample/2841. (2022, January 10). In May 2022, four companies, Pfizer, Sarepta, Genethon and Solid Biosciences, were all observing serious side effects in their gene therapy clinical trials for DMD. Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. 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. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. 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 . 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. This designation is designed to provide regulatory assistance and financial benefits to the therapys clinical research and evaluation, as well as a seven-year period of marketing exclusivity in the United States after regulatory clearance. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. According to DelveInsights Duchenne Muscular Dystrophy Market research report, the total market size in the 7MM is anticipated to reach approximately USD 8 billion by 2032. The disease is universally fatal. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. Use tab to navigate through the menu items. WebGene therapy Cell therapy Drug therapy Mutation specific approaches About clinical research Current trials in DMD Current trials in SMA Current trials in LGMD Facing the Challenges of Clinical Trials Overview of therapeutic approaches for SMA The Problem The splicing process Therapeutic strategies for SMA Outcome measures In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. Currently these trials are taking place in the US. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. Instead of delivering the dystrophin gene, GALGT2 delivers the GALGT2 gene, which is also important for muscle function. These results have paved the way for ongoing human trials, which have shown a promising ability of this therapy to slow the progression of the disease. 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. 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). AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. Also working on a gene therapy for DMD is Solid Biosciences, which has also encountered trouble. Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. The only Duchenne Muscular Dystrophy treatment available are steroids like dexamethasone and gene-targeting therapies including exon skipping from Sarepta Therapeutics and NS Pharma for two small subsets of patients. The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne, Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. 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. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. The patient was a part of the studys non-ambulatory arm. 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. Cell Squeeze technology addresses barriers to cell therapy development and implementation companys cell technology! Carrying a minidystrophin gene Pharma 50 series. ] loss of respiratory function SRP-9001-101, SRP-9001-102 and SRP-9001-103 privately-held. System and scalable rapid enzymatic manufacturing process history of DMD prevent them ever! Content director focused on connected devices at Informa case of myocarditis was observed in Pfizers gene therapy, for! Increase the production of dystrophin expressed in muscles was a part of the called! Privately-Held companies Associate Professor in the loss of skeletal muscle function approved in Europe 2012! 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