The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. It is developed based on exon skipping technology. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. The collaboration could be worth more than $3 billion. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Vyondys 53 (golodirsen) Injection. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. We had been studying in dogs a disease that phenotypically appeared analogous to Duchenne dystrophy for several years going back into the early 80s, said Kornegay. 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. Reference: Barry Byrne, Joe Kornegay, et al., Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Science Translational Medicine (2023), DOI: 10.1126/scitranslmed.abo1815, Feature image: The protein dystrophin. 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. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). There are two main types of gene therapy: somatic gene therapy and germline gene therapy. 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. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Most boys stop walking and need a wheelchair between 9 and 14 years old.. Viltepso is an antisense oliogonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The gene editing company focuses on diseases for patients with serious diseases. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. The company is developing CRISPR/Cas9 genome editing technology. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. Connect with him on LinkedIn or email at [emailprotected], Copyright 2023 WTWH Media LLC. 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. SRP-9001: Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. 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. 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. DelveInsight is a Business Consulting and Market research company, providing expert business The companys AI workbench is finding use for CNS and metabolic disease. 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. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. 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). 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. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle 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). We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. The company is working with the Cas9 and Cas12a CRISPR nucleases. Today, many AAV-based gene therapy medications are Today, many AAV-based gene therapy medications are EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301. One of those labs, Louis Kunkels lab, identified the dystrophin gene first in 1986.. Thankfully, another group of researchers working on a milder form of Duchenne muscular dystrophy called Becker muscular dystrophy found that in these patients that large chunks of the gene were missing but a protein was still produced. Sarepta Therapeutics has two DMD gene therapies, SRP-9001 Micro-dystrophin and GALGT2 (Nationwide Childrens), in clinical trials and one therapy, GNT0004 Micro-dystrophin (Genethon), in preclinical development. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). The American Society of Clinical Oncology is a platform that provides a global connection to researchers, pharma companies, and healthcare professions standing against cancer, finding a cure for it. This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. The factors driving this growth are the newborn screening of DMD, increasing awareness programs, upcoming launches and approvals, and robust pipeline activity in the gene therapy for DMD. Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by involvement of the shoulder muscles. Medical Design and Outsourcing. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. 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. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. 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. However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. DMD starts to show its effects during early childhood. 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. 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. 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. EMDR (801) 436-5597. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. Rocket Pharmaceuticals is aiming for its first regulatory filing in H1 of 2023 for its LVV gene therapy RP-201 for Leukocyte Adhesion Deficiency-I (LAD-I) a rare, autosomal recessive pediatric disease where, without a successful bone marrow transplant. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. You can compare outcomes to natural disease due to a rich natural history of DMD. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. 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. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. Published: Nov 28, 2022 Their first gene therapy product, Zynteglo, was approved by the European Medicines Agency in 2019 to treat a form of inherited anemia. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. Feb 18, 2022 | Reading Time: 8 minutes. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. Sarepta is currently the leading gene therapy player in the DMD space. 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. 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 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. The company boasts that its AI Workbench can help manage the complexity in RNA biology while identifying novel targets. He has extensive research experience in DMD. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. 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. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. 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. All three companies are using different versions of minidystrophin, explained Hesterlee. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. He is currently providing CMC advice for Myosana. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. Pharma50: 50 Leading Cell and gene therapy companies. Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has Significant Limitations; Adeno-Associated Virus (AAV) is a common virus used to deliver "fragments" of healthy genes to the cells that contain unhealthy (mutated) genes. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. The companys allogeneic CAR-T program targets B-cell malignancies. 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. 1985 - 2023 BioSpace.com. That year, Bayer also acquired BlueRock Therapeutics. 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. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. 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. We have developed other critical functions to ensure proper gene delivery. It has six programs in clinical development. 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. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. We know whats wrong, well fix it! Hesterlee added. Solids is different because it contains the binding spot for an enzyme called nitric oxide synthase both Sarepta and Pfizer cut that portion out.. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. A Range of Possible Mechanisms. 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