Web2 Department of Gene Therapy, Saad Pharmaceuticals, Tornime 7-26, Tallinn, 10145, Estonia. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. We know whats wrong, well fix it! Hesterlee added. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. The trials participants will get either a single infusion of gene therapy or a placebo, and they will be tracked for 52 weeks (about a year). Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. 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. 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. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. 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 patient was a part of the studys non-ambulatory arm. 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. SLL is Gilead Buys Out Rights to Cancer Therapy from Jounce for USD 67 Million Gilead Sciences must have liked what it saw in a two-year-old collaboration with Jounce Therapeutics for CCR8-targeting cancer immunotherapy because the company has just agreed to own the program fully. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. Once we identified the culprit gene, we thought Oh great! SGT-001 has received Rare Pediatric Disease and Fast Track Designation in the United States and Orphan Drug Designation in the US and EU in 2017. 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. The trick was using higher doses and the right serotypes of AAV to move the vector out of the bloodstream and into muscle, Hesterlee added. Vyondys 53 (golodirsen) Injection. PF-06939926 was granted Fast Track designation in 2020. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. https://www.pharmalive.com/wp-content/uploads/2021/08/Mega-3-Billion-Deal-Shapes-Up-for-Roche-to-Target-AD-and-Parkinsons-BioSpace-8-24-21.jpeg, https://www.pharmalive.com/wp-content/uploads/2020/01/Pharmalive_4c-300x37.png, FDA accepts BLA for Roche-Sarepta's DMD gene therapy, Copyright - PharmaLive and Outcomes LLC |, Axsome headed to FDA after Phase III Alzheimers agitation win, Social Determinants of Health (SDOH): Three Trends to Watch in 2023, U.S. Centers for Disease Control and Prevention (CDC). They finally found the perfect balance, naming the shortened genes microdystrophins.. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. The collaboration could be worth more than $3 billion. FDA Approves BeiGenes Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. DelveInsight is a Business Consulting and Market research company, providing expert business Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. 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). WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. Sarepta is headquartered in Cambridge, Massachusetts, the US. Cell and gene therapy companies also aim to cure common forms of blindness and restore sight in patients with inherited retinal diseases. The company then opened U.S. enrollment for a Phase III trial of the therapy that was already underway in the U.K., Canada and other countries. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. Published: Nov 28, 2022 This type of gene therapy has the potential to pass the modified genes onto future generations, but it is still experimental and raises ethical concerns. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. Pfizer plans to begin a Phase III study with PF-06939926 by the end of 2020. Gene therapies are a promising treatment option, and a recent study published in Science Translational Medicine describes success with one such therapy using an animal model. But unfortunately, a participant in Pfizers Phase Ib open-label study died unexpectedly. The life span of boys with DMD has been growing steadily (from in their teens to early 30s) due to improvements in heart and respiratory care. The pharma and biotech editor of WTWH Media, Brian is a veteran journalist with more than 15 years of experience covering an array of life science topics, including clinical trials, drug discovery and development and medical devices. Which Countries Top the Chart in Global Pharmaceutical Market? 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. Before coming to WTWH, he served as content director focused on connected devices at Informa. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics 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. 617). A number of pharmaceutical companies are developing drugs and therapies to treat DMD. According to Byrne, these results were used to justify an application for human trials, which are now ongoing. Currently these trials are taking place in the US. The trials main purpose is to monitor changes in NSAA scores. Duchenne muscular dystrophy (DMD) is a rare, fatal Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. According to Kornegay, We showed remarkable decline in loss of respiratory function.. Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). Sarepta and its partner Roche presented new results and analyses on their experimental gene therapy SRP-9001 for the neuromuscular condition Duchenne muscular dystrophy showed consistent, statistically significant functional benefits in individuals. The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Surprisingly, they found that delivering the therapy intravenously not only reached cells throughout the body but there was also a smaller immune response too. 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. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. Gene therapy; Cell therapy; Drug therapy; Mutation specific approaches; TREAT-NMD Services Limited is a wholly owned subsidiary of TREAT-NMD Alliance Limited, a registered charity in England & 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. An IND application is expected by the end of 2021. 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Stan was elected to the Washington State Academy of Sciences in 2013 and selected as a Fellow of the American Association for the Advancement of Sciences in 2017. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). It is developed based on exon skipping technology. 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 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 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. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. He is currently providing CMC advice for Myosana. 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). WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. The company is developing CRISPR/Cas9 genome editing technology. Sarepta Therapeutics. WebAbstract. A Range of Possible Mechanisms. Fixing the mutated gene (through gene editing) or providing cells with a new healthy copy of the gene (through gene therapy) would provide the best benefit, possibly even leading to a lifelong cure. Louise Rodino-Klapac, CSO, executive VP and head of R&D, Sarepta Permission granted by Sarepta If approved, SRP-9001, would be the first gene therapy for the muscular degenerative disease known as DMD and is slated for complete evaluation under the accelerated approval path by the end of May 2023. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Abeona aims to develop therapies for a variety of diseases, including Recessive Dystrophic Epidermolysis Bullosa and Sanfilippo Syndrome Type A. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. 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. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. It has a pipeline of in vivo and ex vivo therapies. But we were cautious after the high profile death of Jesse Gelsinger in 1999.. This button displays the currently selected search type. After almost 15 years since the first gene therapy trial for Duchenne muscular dystrophy (DMD) began, the dream of a DMD gene therapy drug is getting closer to a reality. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310. The company is working with the Cas9 and Cas12a CRISPR nucleases. MDA gave research grants to four labs tasked with finding the cause. PF-06939926was granted Fast Track designation in 2020. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. Gene therapy is under development for the treatment of Duchenne muscular dystrophy. On the other hand, high cost of gene therapies restrains the growth to some extent. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. LPC Intern, CMHC-I. As part of the FDAs 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. It is difficult and costly to manufacture large quantities of AAV. Viruses are very well evolved to get into cells, commented Hesterlee. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. The most troublesome symptoms are breathing difficulties. 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. A fifth company plans to begin dosing in 2023. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. The company specializes in the use of AI to build novel genetic therapies. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Throughout the late 1990s and early 2000s, researchers tinkered with the dystrophin gene, figuring out what parts were needed and how much they could trim out to still have a functional protein. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. The drug in question, GS-1811 (formerl AbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. It is usually observed between the ages of three and six. Mondays BLA acceptance positions Roche and Sarepta as the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. WebThere are many challenges facing the management of DMD patients in the United Arab Emirates and Kuwait and most likely other countries within the Middle East. The Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. 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. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. January 10, 2023 Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease (read more) January 6, 2023 Genethon wishes you a happy new year 2023! Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i 6 min read. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. 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. 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. The company is working with Roche for commercialization outside the country. By Tristan Manalac. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. Connect with him on LinkedIn or email at [emailprotected], Copyright 2023 WTWH Media LLC. For example, Eteplirsen (Exondys 51) is expected to cost patients around US$ 300,000 for a treatment course and the cost of the treatment can go as high as US$ 750,000 annually. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. 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