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. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. While AAV vectors work great for delivering gene therapies to muscle cells, as Barry Byrne, co-author of the new study and professor of pediatrics at the University of Florida, explained, they have a size limitation. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. 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. Contact information and locations are not yet available, but initial trial sites are expected to open in the United States, with sites in Canada and Europe to follow. The companys AI workbench is finding use for CNS and metabolic disease. Microdystrophin expression was seen via muscle biopsies 90 days after treatment (at a dose of 2E14 vg/kg), which stabilized dystrophin-associated proteins and restored activity of a key enzyme (called neuronal nitric oxide synthase, or nNOS) in the muscles. They also have 12 other exon skipping-based genetic medicines in their pipeline. AAV9 is a type of AAV that is particularly good at getting into muscle cells. The Food and Drug Administration approved the therapies after studying a few dozen boys. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. March 29, 2006. 1985 - 2023 BioSpace.com. The collaboration could be worth more than $3 billion. 2020 by Myosana Therapeutics, Inc.. AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. SRP-9001 was safe and well-tolerated up to one-year post-administration. 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). DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. Click for Index The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). 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. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). This is based on a proprietary algorithm built from the drugs sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalDatas Pharmaceutical Intelligence Center. USA: 304 S. Jones Blvd #2432, Las Vegas NV 89107 India: 428, Corporate Park, Sector-21, Dwarka, New Delhi-110077, India, Interested In Knowing The Developments Across Pipeline and Market Forecasts, 304 S. Jones Blvd #2432, Las Vegas NV 89107, 428, Corporate Park, Sector-21, Dwarka, New Delhi-110077, India, Obesity - Market Insight, Epidemiology And Market Forecast - 2032, Gene therapy for duchenne muscular dystrophy, Global Top Players in Intraocular Lens (IOL) Market, How Robots Are Introducing A New Dimension To Healthcare Service Delivery, Analyzing the Most Promising Drugs That Will Lose Patent in the US & EU in 2022. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne of R&D, Strategy Formulation, The disease is universally fatal. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. WebHigh cost of Duchenne muscular dystrophy treatment. 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.. The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. Feb 18, 2022 | Reading Time: 8 minutes. Their first gene therapy product, Zynteglo, was approved by the European Medicines Agency in 2019 to treat a form of inherited anemia. 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. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. However, unlike Sarepta, Pfizer does not have any additional candidates that may join the market and earn market share if its gene therapy treatment fails to win approval, implying that the stakes are higher for the latter. 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. Antibody status can be quite divisive in the DMD community.. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne 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. He is currently providing CMC advice for Myosana. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. Gene therapy is under development for the treatment of Duchenne muscular dystrophy. These DMD therapies may, at best, slow the progression of Duchenne. The field continues to multiply in size. SRP-9001 (2E14 vg/kg dose) is currently being investigated in open-label Phase I/II study (Study 101). Founded in 2014, Intellia Therapeutics is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies for a range of diseases, including cancer and genetic disorders. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. The These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. 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. All Rights Reserved. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. Viruses are very well evolved to get into cells, commented Hesterlee. Take out the viral genes required to make copies of itself and put in the healthy copy of dystrophin, then the virus can get inside cells but not replicate.. 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. 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). AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. FDA accepts BLA for Roche-Sareptas DMD gene therapy. Both employ exon skipping, redirecting DNA processing inside the muscle cells to create minidystrophin right in the cells much like the researchers did in the lab, but directly in the children themselves. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Dystrophin, the largest gene in the human body, encodes a muscle protein responsible for keeping muscle cells from pulling themselves apart when the muscle is working, like a shock absorber for the cell, as Hesterlee described. Now, after serving three years in a Chinese prison for practicing medicine without a license, he faces obstacles and critics as he tries to re-enter science. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. As per DelveInsight Duchenne Muscular Dystrophy Epidemiology Report, the total DMD prevalent population was more than 30K in the 7MM in 2020, which is further expected to increase by 2032. In addition, Brian covered the medical device sector for 10 years at UBM. Details >>, provide genotype and phenotype data from the same cell across thousands of single cells, 25 novel therapies set to shape the landscape of medicine in 2023, Genascence believes gene therapy can transform the treatment of knee osteoarthritis, Drug Discovery & Developments top stories of 2022. 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. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 The companys Tapestri platform can simultaneously. According to data from Solids clinical program, SGT-001 has the potential to slow or stop the Duchenne progression, regardless of genetic mutation or disease stage. WebAbstract. Verified The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. WebDr Paul Benson is an oral and facial surgeon, serial entrepreneur and business coach with a diverse portfolio of companies in a variety of industries including healthcare, beauty, It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.. On the other hand, high cost of gene therapies restrains the growth to some extent. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. 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