Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. 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. 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 & WebHigh cost of Duchenne muscular dystrophy treatment. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. The company is working with the Cas9 and Cas12a CRISPR nucleases. Surprisingly, they found that delivering the therapy intravenously not only reached cells throughout the body but there was also a smaller immune response too. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. It also selectively licenses its NAV vectors to other biotechnology companies. Click for Index 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 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. It is usually observed between the ages of three and six. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. The companys single-course gene editing programs focus on conditions with a genetically driven, life-long and severely elevated LDL-C such as familial hypercholesterolemia (FH). Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. 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. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). The next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. The team has several theories as to why and Byrne believes the issue is solvable. The most troublesome symptoms are breathing difficulties. It has a pipeline of in vivo and ex vivo therapies. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. 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. All rights reserved. They are currently focused on developing gene therapies for a range of diseases, including cancer and genetic disorders. 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. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. Duchenne Muscular Dystrophy is caused by a dystrophin gene mutation (a protein in the muscles). Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. The company previously reported 1-year data for the same measures in March 2021. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. The The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. DMD has a life expectancy of 16 to early 20s. He had previously held managing editor roles on two of the companys medical device technology publications. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. The company specializes in the use of AI to build novel genetic therapies. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. All Rights Reserved. Pfizer plans to begin a Phase III study with PF-06939926 by the end of 2020. 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.. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. 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. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Sarepta Therapeutics. 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. It also has a muscle-specific promoter, which is a DNA element that regulates the activity of a gene called MHCK7. DMD is a progressive muscle wasting disease caused by a genetic mutation. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. By Chelsea Weidman Burke. The approved DMD therapies received a positive response and helped the patients. Verified 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. Their gene therapy product, SB-525, is currently in clinical trials for the treatment of hemophilia A. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. 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. Gene therapies are a promising treatment option, and a recent study published in Science Translational Medicine describes success with one such therapy using an animal model. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. A Range of Possible Mechanisms. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. The FDA has ordered a clinical halt to the trial, and Pfizer is investigating the causes of death. Other hurdles of developing a DMD gene therapy. 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. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. 6 min read. Antibody status can be quite divisive in the DMD community.. They can help slow down disease progression, but tackling inflammation only addresses one downstream effect.. Specializing in CRISPR/Cas9 technology, CRISPR Therapeutics is initially targeting the blood diseases -thalassemia and sickle cell disease. 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. Mondays BLA acceptance positions Roche and Sarepta as the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. 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. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. 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 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 companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. The biotech aims to engineer precision genetic medicine for rare diseases. Without dystrophin, the muscle cells suffer from microtears, leading to their demise and progressive muscle wasting. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. An IND application is expected by the end of 2021. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. These micro-dystrophins might provide only partial improvement of muscle function. 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. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. It is usually observed between the ages of three and six. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. The company sells a variety of instruments and consumables. They are currently developing gene therapies using CRISPR/Cas9 technology. They finally found the perfect balance, naming the shortened genes microdystrophins.. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Also working on a gene therapy for DMD is Solid Biosciences, which has also encountered trouble. 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. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. "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.". 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 Vast improvements have been made in managing patients with DMD, but one stubborn CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. [This feature is a part of 2022s Pharma 50 series.]. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. 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. 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. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. Importantly, there were no serious adverse events (only mild to moderate events). Fixing the mutated gene (through gene editing) or providing cells with a new healthy copy of the gene (through gene therapy) would provide the best benefit, possibly even leading to a lifelong cure. 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 Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. Louise Rodino-Klapac, CSO, executive VP and head of R&D, Sarepta Permission granted by Sarepta If approved, SRP-9001, would be the first gene therapy for the muscular degenerative disease known as DMD and is slated for complete evaluation under the accelerated approval path by the end of May 2023. WebWhilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. 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. 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). Moreover, the companies are hoping that their Duchenne Muscular Dystrophy treatment will slow or even stop disease progression, giving patients a chance to avoid the devastating effects of Duchenne. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company is developing CRISPR/Cas9 genome editing technology. 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. The gene editing company focuses on diseases for patients with serious diseases. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i Gene therapy is under development for the treatment of Duchenne muscular dystrophy. Founded in 1992, bluebird bio is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies for rare genetic diseases and cancer. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Sarepta is headquartered in Cambridge, Massachusetts, the US. Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. 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. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. We have developed several monoclonal antibodies against a specific muscle target protein that is present in both skeletal and cardiac muscle. Following this major safety event, the uncertainty surrounding PF-06939926s future could potentially pave the way for Sareptas continued dominance in the field. Pfizer Inc. Website: www.pfizer.com. They also have 12 other exon skipping-based genetic medicines in their pipeline. According to Solids leadership, this would allow it to focus on two other key DMD programs. He is currently a Professor of Physiology and Biophysics at the University of Washington. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. 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. The Food and Drug Administration approved the therapies after studying a few dozen boys. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). of R&D, Strategy Formulation, Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. Cellectis has more than two decades of experience in gene editing. USA: 304 S. Jones Blvd #2432, Las Vegas NV 89107 India: 428, Corporate Park, Sector-21, Dwarka, New Delhi-110077, India, Interested In Knowing The Developments Across Pipeline and Market Forecasts, 304 S. Jones Blvd #2432, Las Vegas NV 89107, 428, Corporate Park, Sector-21, Dwarka, New Delhi-110077, India, Obesity - Market Insight, Epidemiology And Market Forecast - 2032, Gene therapy for duchenne muscular dystrophy, Global Top Players in Intraocular Lens (IOL) Market, How Robots Are Introducing A New Dimension To Healthcare Service Delivery, Analyzing the Most Promising Drugs That Will Lose Patent in the US & EU in 2022. 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. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. While Solid Biosciences SGT-001 and Regenxbios RGX-202 are in the early stage of development for DMD treatment. It is administered by intravascular and intramuscular routes. 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 The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Which Countries Top the Chart in Global Pharmaceutical Market? Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. According to Byrne, these results were used to justify an application for human trials, which are now ongoing. Specializes in developing next-generation AAV capsids for gene therapies. The company develops its pipeline products using its multi-platform SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. Likelihood of Approval and Phase Transition Success Rate Mod IMT-009 by Immunitas Therapeutics for Squamous Non-Small Cell Lung Cancer: Likelihood of Approval, IMT-009 by Immunitas Therapeutics for Diffuse Large B-Cell Lymphoma: Likelihood of Approval, IMS-001 by Imstem Biotechnology for Relapsing Remitting Multiple Sclerosis (RRMS): Likelihood of Approval, IMS-001 by Imstem Biotechnology for Primary Progressive Multiple Sclerosis (PPMS): Likelihood of Approval. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne The DMD Gene Therapy Race Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory 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. The company has scored approvals for several RNAi therapeutics. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. 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. REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). Cell and gene therapies promise to enable broad changes in the healthcare system over the next decade, prompting a growing number of cell and gene therapy companies to join the space. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. 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. The collaboration could be worth more than $3 billion. The two nucleases give it access to a variety of genetic mutations and develop targeted and durable gene edited medicines. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. 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, The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. The trials main purpose is to monitor changes in NSAA scores. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. 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. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. 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. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Feb 18, 2022 | Reading Time: 8 minutes. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. The disease is universally fatal. Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. Is investigating the causes of death immuno-oncology and stem cell clinical trials in China with products from its integrated laboratory. I trial is not specifically targeted to muscle, so high doses are required to achieve delivery the! Provide only partial improvement of muscle function now ongoing DMD uses selected portions of participants... 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