As a result, SRP-9001 would gain a competitive edge. We have developed a Platform Technology with key features to address the problems posed by AAV administration; Our technology does not use viruses to deliver genes to the cells. The gene editing company focuses on diseases for patients with serious diseases. Focuses on allogeneic placental-derived cells. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. Sarepta is currently the leading gene therapy player in the DMD space. RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381. Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. The most common type of vector is a virus called adeno-associated virus (AAV), which works using the natural ability of viruses to enter cells and hijack cellular machinery to produce viral proteins encoded by viral genes. 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 FDA accepts BLA for Roche-Sareptas DMD gene therapy. CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. It is difficult and costly to manufacture large quantities of AAV. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. Corticosteroids help dampen down inflammation, said Hesterlee. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? Allied Market Research (AMR) is a full-service market research and business-consulting wing of Allied Analytics LLP based in Portland, Oregon. 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. EMDR (801) 436-5597. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. The patient was a part of the studys non-ambulatory arm. Focusing on developing therapeutics for disorders of the central nervous system. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. The company is using a stem cell biology and genomics platform to develop a novel autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy for Parkinsons Disease. It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. Solids is different because it contains the binding spot for an enzyme called nitric oxide synthase both Sarepta and Pfizer cut that portion out.. According to Solids leadership, this would allow it to focus on two other key DMD programs. Or higher doses to drive the virus into the muscles? Hesterlee added. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. Pfizer Inc. Website: www.pfizer.com. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. DMD is a progressive muscle wasting disease caused by a genetic mutation. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. solutions for life science vertical and offering quintessential advisory services in the Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. 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. 6 min read. The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. Importantly, our platform is Not Restricted By Gene Size. Allied Market Research provides global enterprises as well as medium and small businesses with unmatched quality of Market Research Reports and Business Intelligence Solutions. AMR has a targeted view to provide business insights and consulting to assist its clients to make strategic business decisions and achieve sustainable growth in their respective market domain. PF-06939926 was granted Fast Track designation in 2020. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. The company is working with Roche for commercialization outside the country. This may lead to dangerous side effects. 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. Thats why the first DMD gene therapy trial in the US, which began in 2006, involved injecting the gene therapy directly into the biceps of the children who participated. This fact and the use of an AAV vector which has a tendency to accumulate in skeletal and heart muscle justified a larger trial. 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. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. As part of the FDA's 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. You can compare outcomes to natural disease due to a rich natural history of DMD. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. 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. 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. 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. 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. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. 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. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i This button displays the currently selected search type. 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. 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. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. 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. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. 1985 - 2023 BioSpace.com. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). At Qmed, he overhauled the brands news coverage and helped to grow the sites traffic volume dramatically. A gene transfer therapy study to evaluate the safety and efficacy of SRP-9001 in participants with Duchenne Muscular Dystrophy (DMD) [NCT05096221]. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. Buy the report here. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. We are able to directly target the muscle cells. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. All rights reserved. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Most boys stop walking and need a wheelchair between 9 and 14 years old.. The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) 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). A number of pharmaceutical companies are developing drugs and therapies to treat DMD. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Also working on a gene therapy for DMD is Solid Biosciences, which has also encountered trouble. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Data are expected to start rolling in late next year. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Eventually, they will need ventilation to help them breathe. 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. The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. https://www.alliedmarketresearch.com/request-sample/2841. 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. Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. 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. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. 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