dmd gene therapy companies

Their gene therapy product, SB-525, is currently in clinical trials for the treatment of hemophilia A. RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381. WebThe Roche Groups bold commitment to gene therapy collaborations across the organisation and industry are a stake in the ground: the possibility and potential of using 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). Rare Daily Staff. 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. EMDR (801) 436-5597. The company was spun-out from University College London in 2014. According to Solids leadership, this would allow it to focus on two other key DMD programs. Gene therapy replaces the mutated gene with a copy using whats called a vector to bring a working copy of the gene into a cell. Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the body's immune responses to the protein expressed by their gene therapeutic. The company sells a variety of instruments and consumables. Currently these trials are taking place in the US. 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 goal of gene therapy is to replace or repair a missing or faulty gene, introduce a new gene to help fight disease, or deactivate a harmful gene. The There are currently four companies who have DMD gene therapy products that have been given to boys with Duchenne, and three of the companies have ongoing studies in the USA. Once we identified the culprit gene, we thought Oh great! AAV is most often restricted to a single administration due to the likelihood of an immune response to the virus. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). Verified 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. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). Pharmaceutical companies see the value too, with one company, Sarepta, expecting approval of a Duchenne muscular dystrophy gene therapy as early as June of this year. The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. Cellectis has more than two decades of experience in gene editing. 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. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. It is administered by intravascular and intramuscular routes. What is now called golden retriever muscular dystrophy (GRMD) turns out to be an excellent model for Duchenne muscular dystrophy because it involves the canine dystrophin gene, causes similar symptoms, and is also X-linked. Each of these gene therapies has slight variations in their three main components: the transgene, the 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 . Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. 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. The mutated gene is on the X chromosome, making DMD an X-linked disease. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. 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. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. 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. The FDA soon put the Phase Ib study under clinical hold. The company also has a collaboration with BioMarin. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. 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. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. 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. Importantly, our platform is Not Restricted By Gene Size. 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). 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. According to the companys press release, preliminary data from nine boys with DMD (ages 6-12) showed the therapy was well-tolerated during intravenous infusion. 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. NIH, U.S. National Library of Medicine, ClinicalTrials.gov. 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. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. DMD is a progressive muscle wasting disease caused by a genetic mutation. Gene therapy; Cell The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. 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. 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. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. The problem is exon skipping, in its current form, is not very efficient and each therapy only works in a subset of children with certain gene mutations, Hesterlee commented. The trials main purpose is to monitor changes in NSAA scores. We are able to directly target the muscle cells. 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. Focuses on clinical-stage gene therapy. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9), being developed by Asklepios BioPharmaceutical (AskBio). But the disease doesnt just affect their legs it affects muscles all over their body. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. BioSpace sat down with Sharon Hesterlee, Ph.D., chief research officer at the Muscular Dystrophy Association (MDA), to talk about the history and challenges of developing gene therapy for DMD and the DMD gene therapy field as a whole, including Pfizers and Sarepta Therapeutics latest clinical data. 1. We have developed several monoclonal antibodies against a specific muscle target protein that is present in both skeletal and cardiac muscle. This would appear to be an easy solution. The Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. 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