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 That worked great for small genes, but not so well for dystrophin.. All rights reserved. We dont know exactly why the dog did not predict this severe adverse event, said Kornegay. Gene therapy offers a potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy. 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. 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WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Clinical researchers at UC Davis Health are using a gene therapy approach for Duchenne muscular dystrophy (DMD), the rare genetic disease that mainly occurs in 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). DelveInsight is a Business Consulting and Market research company, providing expert business It employs a non-lethal modified virus (AAVrh74) with a high affinity for muscle tissue, allowing for targeted delivery. 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. However, gene therapy for Duchenne muscular dystrophy still has several hurdles to overcome. 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. Viruses are very well evolved to get into cells, commented Hesterlee. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. 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. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. 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 clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. The mutated gene is on the X chromosome, making DMD an X-linked disease. Byrne and colleagues now had a therapeutic that would fit in the AAV. A number of companies are now testing their approaches in the clinic. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. 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. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. Back in the mid-1980s, the cause of DMD was still unknown all we knew was that it ran in families, there were no genes associated with the disease yet, Hesterlee explained. In patients with Duchenne muscular dystrophy, the affected gene codes for the protein, dystrophin, which acts as a shock absorber between muscle cells and connective tissue, as well as supporting muscle contraction. Before coming to WTWH, he served as content director focused on connected devices at Informa. 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 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. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. 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. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. According to the market research firm Emergent Research, international cell and gene therapy companies could generate $6.6 billion in revenue by 2027, with a projected CAGR of 19.8% from 2020 to 2027. WebThe Roche Groups bold commitment to gene therapy collaborations across the organisation and industry are a stake in the ground: the possibility and potential of using They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. Get industry leading news, data and analysis delivered to your inbox. The company is working with Roche for commercialization outside the country. SRP-9001 was safe and well-tolerated up to one-year post-administration. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. 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. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310. Sarepta is responsible for SRP-9001's U.S. application. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. 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. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. 617). The trials main purpose is to monitor changes in NSAA scores. They are currently developing gene therapies using CRISPR/Cas9 technology. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. The FDA soon put the Phase Ib study under clinical hold. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. All rights reserved. The FDA hasacceptedRocheandSareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD). Allied Market Research (AMR) is a full-service market research and business-consulting wing of Allied Analytics LLP based in Portland, Oregon. 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.. 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