This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. The disease is universally fatal. They are currently developing gene therapies using CRISPR/Cas9 technology. They are currently focused on developing gene therapies for a range of diseases, including cancer and genetic disorders. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. 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. 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. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? 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. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. 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. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. The companys allogeneic CAR-T program targets B-cell malignancies. 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 But there is a limit to how much cargo you can stuff inside these tiny viruses, about 5 kb for AAV. 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. March 29, 2006. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. MDA gave research grants to four labs tasked with finding the cause. Duchenne muscular dystrophy (DMD) is a fatal condition caused by a single gene mutation on the X-chromosome being X-linked means only males suffer Rare Daily Staff. The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. We have developed other critical functions to ensure proper gene delivery. However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). Instead of delivering the dystrophin gene, GALGT2 delivers the GALGT2 gene, which is also important for muscle function. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. Importantly, there were no serious adverse events (only mild to moderate events). 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. Published: Nov 28, 2022 The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. 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. LPC Intern, CMHC-I. solutions for life science vertical and offering quintessential advisory services in the Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. The approved DMD therapies received a positive response and helped the patients. The disease is universally fatal. The FDA soon put the Phase Ib study under clinical hold. Other hurdles of developing a DMD gene therapy. We know whats wrong, well fix it! Hesterlee added. 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. Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. These micro-dystrophins might provide only partial improvement of muscle function. 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. 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. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. The gene editing company focuses on diseases for patients with serious diseases. Specializes in developing next-generation AAV capsids for gene therapies. Knowing your family history is the first step to understand and be proactive about your Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. In September 2021, Astellas Pharma reported a fourth patient death in the AT132 clinical Gene therapy is under development for the treatment of Duchenne muscular dystrophy. The company has scored approvals for several RNAi therapeutics. Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. But it took another 30 years to be able to apply this knowledge to develop effective drugs., Although corticosteroids can slow the progression of DMD to some extent, they dont address the underlying issue the lack of functional dystrophin. Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. The company previously reported 1-year data for the same measures in March 2021. 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. Vyondys 53 (golodirsen) Injection. The companys core focus areas include immuno-oncology and plant sciences. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. In September 2021, the company announced. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Terry Horgan, the primary patient in an N-of-1 clinical trial evaluating a CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has died, according to an announcement from Cure Rare Disease, the nonprofit biotech sponsoring the trial. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. 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. 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. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Byrne and colleagues now had a therapeutic that would fit in the AAV. As a result, SRP-9001 would gain a competitive edge. Get industry leading news, data and analysis delivered to your inbox. 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. Extensive pre-clinical evidence also formed part of the BLA. Web2 Department of Gene Therapy, Saad Pharmaceuticals, Tornime 7-26, Tallinn, 10145, Estonia. Krystal Biotech specializes in redosable gene therapy. It is administered by intravascular and intramuscular routes. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. All rights reserved. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). Medical Design and Outsourcing. In fact, the FDA recently granted SRP-9001 Fast Track designation. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). of R&D, Strategy Formulation, The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. But unfortunately, a participant in Pfizers Phase Ib open-label study died unexpectedly. Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. The first signs of DMD appear as the young boys begin to walk and get more mobile, typically between the ages of 2 to 5. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. 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. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. 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. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. For example, the dystrophin gene is too large to fit into the adeno-associated viruses, or AAVs, that are commonly used to deliver gene therapies. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. 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, anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. The trials main purpose is to monitor changes in NSAA scores. Focuses on clinical-stage gene therapy. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. 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. 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. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. Back in the mid-1980s, the cause of DMD was still unknown all we knew was that it ran in families, there were no genes associated with the disease yet, Hesterlee explained. 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. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. Founded in 1995, Sangamo Therapeutics is a biotech company based in Richmond, California that focuses on developing gene therapies for rare genetic diseases and cancer. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. 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. 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 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. Most boys stop walking and need a wheelchair between 9 and 14 years old.. 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. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. 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). In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. 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. Sarepta is headquartered in Cambridge, Massachusetts, the US. 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. Monkel is an established researcher in the neuromuscular disease field and his research has led to novel gene discoveries for previously undiagnosed rare muscle diseases. The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. 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. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. The company was spun-out from University College London in 2014. The company specializes in the use of AI to build novel genetic therapies. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715. This not only quickly diminishes the amount of therapeutic virus in the body, but it could also mean the patient would only be able to get one dose of therapy - any subsequent doses would be destroyed too quickly by the body or, worse, potentially cause a severe immune reaction. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. 1985 - 2023 BioSpace.com. 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. 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 & Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. Data are expected to start rolling in late next year. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. 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. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. 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. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. Currently these trials are taking place in the US. The companys AI workbench is finding use for CNS and metabolic disease. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. 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). In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. Sometimes called minidystrophins, there are slight variations between different versions of these shortened genes, but the key is they are all small enough to fit into AAV, explained Hesterlee. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. The biotech is developing novel cell and exosome-based therapeutics. 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 companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. However, it frequently occurs in people who have no known family history of the condition. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. From heart or respiratory failure a number of pharmaceutical companies are also to... Benefit, said Byrne size of the global Duchenne Muscular Dystrophy ( DMD Core. An immune response $ 1.15 billion upfront for exclusive rights to SRP-9001 the Tabelecleucel ( tab-cel ) ATA188. Ntla-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001 AUTO6NG, AUTO8 to target muscle tissue, resulting the... System side effects in humans, with Roche surrendering $ 1.15 billion upfront for exclusive to! 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