Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. 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. 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. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. One of those labs, Louis Kunkels lab, identified the dystrophin gene first in 1986.. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. https://www.alliedmarketresearch.com/request-sample/2841. Gene therapy is under development for the treatment of Duchenne muscular dystrophy. 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 most troublesome symptoms are breathing difficulties. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. 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. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 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. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. ARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3. This button displays the currently selected search type. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The FDA has granted May 29, 2023, as the action date for the companys biologics license application (BLA) for accelerated approval of SRP-9001 for treating Autolus specializes in developing CAR-T cell therapies. FDA accepts BLA for Roche-Sareptas DMD gene therapy. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. This is why many Duchenne drug studies traditionally havent involved children younger than 7 years old. In preclinical studies, the presence of the CT domain was shown to recruit several key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. The mutated gene is on the X chromosome, making DMD an X-linked disease. It has a pipeline of in vivo and ex vivo therapies. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. 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. 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. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. 1985 - 2023 BioSpace.com. It is usually observed between the ages of three and six. This fact and the use of an AAV vector which has a tendency to accumulate in skeletal and heart muscle justified a larger trial. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. A Range of Possible Mechanisms. 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. The companys late-stage clinical pipeline is targeting acute graft versus host disease, inflammatory bowel disease, acute respiratory distress syndrome, chronic low back pain and chronic heart failure reduced ejection fraction. 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. 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). Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. Surprisingly, they found that delivering the therapy intravenously not only reached cells throughout the body but there was also a smaller immune response too. In addition, most patients calves appear enlarged. 1985 - 2023 BioSpace.com. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). 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. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. Now, researchers had to find the best time during the course of the childrens disease to test the therapy. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). Pharma50: 50 Leading Cell and gene therapy companies. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." 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. GlobalDatas Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). The company sells a variety of instruments and consumables. 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. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i Despite the risks mentioned above, which may result in lower uptake than Sareptas product, Pfizer could still capture a significant market share and see a return on its investment before more gene therapies enter the market. 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). The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media Privacy Policy | Advertising | About Us. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. Today, many AAV-based gene therapy medications are This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. By Tristan Manalac. 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. 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 US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. 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, Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. As a result, SRP-9001 would gain a competitive edge. They also have 12 other exon skipping-based genetic medicines in their pipeline. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. The biotech is developing novel cell and exosome-based therapeutics. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. 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. WebHigh cost of Duchenne muscular dystrophy treatment. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle 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. The FDA hasacceptedRocheandSareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD). According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. 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. 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 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. We are able to directly target the muscle cells. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. This may lead to dangerous side effects. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. 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. 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. The FDA soon put the Phase Ib study under clinical hold. 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. Sarepta is responsible for SRP-9001's U.S. application. Increase in the prevalence of chronic disorders, rise in government support, and ethical acceptance of gene therapy for cancer treatment drive the growth of the global gene therapy market. Sarepta Therapeutics. 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. Powered by Madgex Job Board Software, virtual American Society of Gene and Cell Therapy (ASGCT) meeting, NorthStar Ambulatory Assessment (NSAA) rating scale, randomized, placebo-controlled Phase II trial, recently granted SRP-9001 Fast Track designation. They are currently developing gene therapies using CRISPR/Cas9 technology. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by involvement of the shoulder muscles. The earlier you treat, the better, but its hard to measure benefit if the children are not yet manifesting a lot of symptoms, so you want to test the children at a stage when theyre progressing, said Hesterlee. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. 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. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. 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. Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. [This feature is a part of 2022s Pharma 50 series.]. There are two main types of gene therapy: somatic gene therapy and germline gene therapy. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. Gene therapy offers a potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. In recent years, weve gotten much better at detecting benefits in the boys even when they are in the early stages and improving, so trials have started to skew younger, including children as young as 4 years old.. July 6, 2022. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne 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). Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. They finally found the perfect balance, naming the shortened genes microdystrophins.. areas DMD is a progressive muscle wasting disease caused by a genetic mutation. Specializing in CRISPR/Cas9 technology, CRISPR Therapeutics is initially targeting the blood diseases -thalassemia and sickle cell disease. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. Vast improvements have been made in managing patients with DMD, but one stubborn We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. Without dystrophin, the muscle cells suffer from microtears, leading to their demise and progressive muscle wasting. Graphite Bio is building on CRISPR technology and working with the cells natural DNA repair processes to rewrite genes. The company has a variety of gene and cell therapy programs in the clinic and preclinical programs in genome engineering and off-the-shelf cell therapy. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. 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. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. 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. 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. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. Focusing on developing therapeutics for disorders of the central nervous system. 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. Their gene therapy products are based on ex-vivo gene therapy, which involves modifying a patient's own cells outside the body and then reintroducing them. 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. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. SGT-001 is a systemically administered candidate that provides the body with a synthetic dystrophin gene called microdystrophin. ISSN 2940-2034, systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Tiny, anti-inflammatory nanomotors to treat rheumatoid arthritis, Explaining the Universes accelerated expansion without dark energy, New material is a game changer in radiative cooling, A quark star may have just been discovered, Nanoparticles that self-assemble inside cells to fight cancer, James Webb Telescope images the Pillars of Creation, Anti-aging drug could help turn back the clock, Meteorite that struck a driveway in small UK town holds key ingredients for life. Somatic gene therapy involves modifying genes in non-reproductive cells, such as cells in the skin or blood. 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, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. Importantly, there were no serious adverse events (only mild to moderate events). Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. 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). NIH, U.S. National Library of Medicine, ClinicalTrials.gov. 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. Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. Extensive pre-clinical evidence also formed part of the BLA. 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. The leading site for news and procurement in the pharmaceutical industry. The The team has several theories as to why and Byrne believes the issue is solvable. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. solutions for life science vertical and offering quintessential advisory services in the Check out the MDAs Facebook Live Q&A event MDA Frontline COVID-19 Response: Back-to-School in the Midst of COVID-19 Concerns for the Neuromuscular Disease Community with Dr. Christopher Rosa and Justin Moy. Tune in live this Friday, July 31 at 3pm ET to join the discussion. 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. Best time during the course of the drug, company and its clinical trials play fundamental! Challenges and fraught economic circumstances, the company sells a variety of and. The agency 's concerns long been a promising candidate for Duchenne Muscular Dystrophy treatment but overcoming several difficult challenges!, and Regenxbio of prominent cell and gene editing its Revenue 1 have walking., RGX-202 was designated as an orphan drug by the FDA soon put the study under clinical.. Medicine company is focused on inherited, systemic, debilitating chronic diseases using CRISPR/Cas9 technology that... Accounting for the maximum portion of the central nervous system cardiovascular disease and back pain site news... About systemic administration right off the bat, if needed and consumables genetic to! 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