They also have 12 other exon skipping-based genetic medicines in their pipeline. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). It is very likely that one or both of these gene therapies could be approved., This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation, Hesterlee concluded. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. "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 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 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. Focusing on developing therapeutics for disorders of the central nervous system. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. This button displays the currently selected search type. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. As an example, Dystrophin, the gene responsible for Duchenne muscular dystrophy (DMD) is 14 kb, meaning that only one-third of the dystrophin gene can be "packaged" into AAV. 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. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. Extensive pre-clinical evidence also formed part of the BLA. Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. Rocket Pharmaceuticals is aiming for its first regulatory filing in H1 of 2023 for its LVV gene therapy RP-201 for Leukocyte Adhesion Deficiency-I (LAD-I) a rare, autosomal recessive pediatric disease where, without a successful bone marrow transplant. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. 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. Sarepta Therapeutics. Pfizers PF-06939926 was designated as an Orphan Drug and Pediatric Rare Disease by the FDA in May 2017 and an Orphan Medicinal Product Designation by the EMA for the treatment of DMD. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. It also has a muscle-specific promoter, which is a DNA element that regulates the activity of a gene called MHCK7. 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. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. 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. It also selectively licenses its NAV vectors to other biotechnology companies. Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. The leading site for news and procurement in the pharmaceutical industry. 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. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. In preclinical studies, the presence of the CT domain was shown to recruit several key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle damage in dystrophic mice. By Chelsea Weidman Burke. He has extensive research experience in DMD. 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). AAV9 is a type of AAV that is particularly good at getting into muscle cells. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. Summer Zemp. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. In addition, most patients calves appear enlarged. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. Published: Nov 28, 2022
With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. The biotech is developing novel cell and exosome-based therapeutics. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Or higher doses to drive the virus into the muscles? Hesterlee added. 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. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. DelveInsight is a Business Consulting and Market research company, providing expert business Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. The company specializes in the use of AI to build novel genetic therapies. Viruses are very well evolved to get into cells, commented Hesterlee. 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. SLL is Gilead Buys Out Rights to Cancer Therapy from Jounce for USD 67 Million Gilead Sciences must have liked what it saw in a two-year-old collaboration with Jounce Therapeutics for CCR8-targeting cancer immunotherapy because the company has just agreed to own the program fully. ORLANDO, FloridaJeffrey Chamberlain, PhD, outlined the 4 different types of gene therapy for treating Duchenne muscular dystrophy (DMD) at the Gene Therapy and Gene Editing Symposium which took place on the second day of the CureDuchenne 2022 FUTURES National Conference . 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. 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. 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. Anywhere from 10 to 80 percent of DMD patients, depending on the serotype in question, have preexisting antibodies against AAVs, meaning they are not eligible for gene therapy, Hesterlee elaborated. Viltepso is an antisense oliogonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Sarepta is currently the leading gene therapy player in the DMD space. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. The disease is universally fatal. 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 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. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. As a result, SRP-9001 would gain a competitive edge. Powered by Madgex Job Board Software. The trials participants will get either a single infusion of gene therapy or a placebo, and they will be tracked for 52 weeks (about a year). The biotech specializes in creating gene therapies for severe genetic disorders and cancer. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. The team has several theories as to why and Byrne believes the issue is solvable. What about a tourniquet and pressure? PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. 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 It is currently being investigated in a Phase I/II study in six boys ages 4 and up. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. The companys Tapestri platform can simultaneously. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. Duchenne Muscular Dystrophy is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. Focuses on clinical-stage gene therapy. 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. Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. While Solid Biosciences SGT-001 and Regenxbios RGX-202 are in the early stage of development for DMD treatment. They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. 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 Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023.
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