The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. 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). Sarepta and its partner Roche presented new results and analyses on their experimental gene therapy SRP-9001 for the neuromuscular condition Duchenne muscular dystrophy showed consistent, statistically significant functional benefits in individuals. As a result, SRP-9001 would gain a competitive edge. solutions for life science vertical and offering quintessential advisory services in the Once we identified the culprit gene, we thought Oh great! The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. 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. 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. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. Published: Jul 29, 2020 January 10, 2023 Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease (read more) January 6, 2023 Genethon wishes you a happy new year 2023! Could we use histamine? Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. AAV9 is a type of AAV that is particularly good at getting into muscle cells. The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. Both employ exon skipping, redirecting DNA processing inside the muscle cells to create minidystrophin right in the cells much like the researchers did in the lab, but directly in the children themselves. Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. In this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. 1985 - 2023 BioSpace.com. 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. 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. All rights reserved. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock late last year after a treated patient died. Following this major safety event, the uncertainty surrounding PF-06939926s future could potentially pave the way for Sareptas continued dominance in the field. 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. ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran). He has extensive research experience in DMD. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. 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 gene editing company focuses on diseases for patients with serious diseases. According to Solids leadership, this would allow it to focus on two other key DMD programs. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. 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. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be By Chelsea Weidman Burke. The factors driving this growth are the newborn screening of DMD, increasing awareness programs, upcoming launches and approvals, and robust pipeline activity in the gene therapy for DMD. He had previously held managing editor roles on two of the companys medical device technology publications. 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. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. However, gene therapy for Duchenne muscular dystrophy still has several hurdles to overcome. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. 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. SRP-9001: Focuses on developing novel gene therapies for rare inherited genetic diseases. Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. A Range of Possible Mechanisms. It has six programs in clinical development. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. "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.". Duchenne Muscular Dystrophy is caused by a dystrophin gene mutation (a protein in the muscles). MDA gave research grants to four labs tasked with finding the cause. Regardless, Pfizer will need to examine the situation and acquire the data necessary to continue the Phase Ib trial and make changes to future trials, such as omitting certain mutation types. The disease is universally fatal. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. 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. WebMyosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure to improve the quality of life, increase longevity and reduce the disease burden of Duchenne muscular dystrophy (DMD) and Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. Sarepta is responsible for SRP-9001's U.S. application. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. 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. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. 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. In addition, Brian covered the medical device sector for 10 years at UBM. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. 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. The collaboration could be worth more than $3 billion. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. Email (801) 436-5597. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. 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 Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? The company has multiple cell therapy candidates in pre-clinical and clinical development. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. 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. Rare Daily Staff. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. 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. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. 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. The companys allogeneic CAR-T program targets B-cell malignancies. Participants in Part 2 of Study SRP-9001-102 scored 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) 48 weeks after SRP-9001 treatment compared to a pre-specified matched external control cohort (p value=0.0009). PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. 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. Or higher doses to drive the virus into the muscles? Hesterlee added. 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. Corticosteroids help dampen down inflammation, said Hesterlee. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. It also selectively licenses its NAV vectors to other biotechnology companies. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. Viruses are very well evolved to get into cells, commented Hesterlee. 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. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. In September 2021, the company announced. Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. 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 core focus areas include immuno-oncology and plant sciences. One component condenses the DNA of the gene and protects it from degradation. Many researchers and companies are looking into alternate delivery systems that could transfer the dystrophin gene to the body without using AAVs. 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. Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. Krystal Biotech specializes in redosable gene therapy. That worked great for small genes, but not so well for dystrophin.. Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. SRP-9001 is also being studied in a randomized, placebo-controlled Phase II trial (Study 102) in 41 boys ages 4-7 years with results expected in early 2021. 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