The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. 2020 by Myosana Therapeutics, Inc.. PF-06939926was granted Fast Track designation in 2020. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. 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. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. 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. 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. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. You can compare outcomes to natural disease due to a rich natural history of DMD. 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. 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 The company develops its pipeline products using its multi-platform For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. 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. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). FDA Approved: December 12, 2019; Company: Sarepta Therapeutics 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. Sarepta is headquartered in Cambridge, Massachusetts, the US. 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. As a result, SRP-9001 would gain a competitive edge. 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 Instead of delivering the dystrophin gene, GALGT2 delivers the GALGT2 gene, which is also important for muscle function. 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. 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. Muscle weakness and atrophy spread from the trunk and forearms to other muscles throughout the body as the disease advances. By Tristan Manalac. Its pipeline product includes SRP-5051, SRP-9001, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. 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. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. "The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne," Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. 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. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. 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. It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. 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. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Focusing on developing therapeutics for disorders of the central nervous system. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? According to Byrne, these results were used to justify an application for human trials, which are now ongoing. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). The two nucleases give it access to a variety of genetic mutations and develop targeted and durable gene edited medicines. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the body's immune responses to the protein expressed by their gene therapeutic. 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. 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 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. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. 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. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Pharma50: 50 Leading Cell and gene therapy companies. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. 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. 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. In September 2021, Astellas Pharma reported a fourth patient death in the AT132 clinical Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. Pfizer Inc. Website: www.pfizer.com. The mutated gene is on the X chromosome, making DMD an X-linked disease. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. 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. It is developed based on exon skipping technology. 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. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. Extensive pre-clinical evidence also formed part of the BLA. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. 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). Krystal Biotech specializes in redosable gene therapy. Data are expected to start rolling in late next year. The patients body will react to the viral vector just like it would any other virus, creating antibodies to hunt and destroy the gene therapy viruses. 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. Gene therapy offers a potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? Take out the viral genes required to make copies of itself and put in the healthy copy of dystrophin, then the virus can get inside cells but not replicate.. 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. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. When expanded it provides a list of search options that will switch the search inputs to match the current selection. 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. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. It also has a muscle-specific promoter, which is a DNA element that regulates the activity of a gene called MHCK7. One component condenses the DNA of the gene and protects it from degradation. 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. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. This would appear to be an easy solution. We know whats wrong, well fix it! Hesterlee added. 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. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. (2022, January 10). However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry. Today, many AAV-based gene therapy medications are 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). The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. Autolus specializes in developing CAR-T cell therapies. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. He is currently providing CMC advice for Myosana. The FDA soon put the Phase Ib study under clinical hold. 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. Gene therapy is more efficient and covers everyone, regardless of genetic mutations, but its still good to have options while new therapies are in development.. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne Byrne and colleagues now had a therapeutic that would fit in the AAV. 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. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. solutions for life science vertical and offering quintessential advisory services in the Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. DMD starts to show its effects during early childhood. Graphite Bio is building on CRISPR technology and working with the cells natural DNA repair processes to rewrite genes. The company is working with Roche for commercialization outside the country. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. The company sells a variety of instruments and consumables. The company has scored approvals for several RNAi therapeutics. 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 clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. He is currently a Professor of Physiology and Biophysics at the University of Washington. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. Buy the report here. The disease is universally fatal. 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). But unfortunately, a participant in Pfizers Phase Ib open-label study died unexpectedly. They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. 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 The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. The disease is universally fatal. It also selectively licenses its NAV vectors to other biotechnology companies. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne, Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. The Food and Drug Administration approved the therapies after studying a few dozen boys. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. Focuses on clinical-stage gene therapy. On the other hand, high cost of gene therapies restrains the growth to some extent. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. Horgan is the brother of Cure Rare Disease founder Rich Horgan, Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. Antibody status can be quite divisive in the DMD community.. 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. Even if both gene therapies reach the market, PF-06939926 is likely to face a delay due to the recent death in its Phase Ib trial. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. Focuses on developing novel gene therapies for rare inherited genetic diseases. The NAV AAV8 vector, which has been used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12) are used in RGX-202 to support the delivery and targeted expression of genes throughout skeletal and heart muscle. By Chelsea Weidman Burke. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. "Within the context of Duchenne and other rare diseases, it's a very robust sample size and one that will grow with data from EMBARK.". GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. Gene therapy replaces the mutated gene with a copy using whats called a vector to bring a working copy of the gene into a cell. The companys NEXI-001 and NEXI-002 programs are in Phase 1/2 clinical studies for relapsed acute myeloid leukemia after allogeneic stem cell transplantation and multiple myeloma refractory to >3 prior lines of therapy, respectively. 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. WebDuchenne muscular dystrophy is the result of a mutation or deletion of the gene encoding dystrophin, located on the X chromosome. Summer Zemp. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. 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. All rights reserved. He is currently the Scientific Founder and SAB Chairman of Bolden Therapeutics. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. 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. Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. Pfizer Inc. Website: www.pfizer.com. The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. Potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy and hemophilia restrains growth. 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