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Jun 27, 2023 · Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy Nov 28, 2022 Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the ... Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... Jul 22, 2022 · Currently, four notable gene therapy candidates are under development: PF-06939926 from Pfizer, SRP-9001 from Sarepta Therapeutics, SGT-1001 from Solid Biosciences, and GNT 0004 from Genethon. There are two Ph3 trials registered at ClinicalTrials.gov to evaluate PF-06939926 and SRP-9001. These trials have an enrollment size of 99–120, and the ... Apr 22, 2021 · SRP-9001 is an adeno-associated virus (AAV) mediated gene therapy that delivers a micro-dystrophin-encoding gene to the muscles and is intended as a curative treatment. The specific vector deployed in the gene transfer, AAVrh74, has been shown to achieve the efficient delivery of micro-dystrophin to skeletal muscle with tolerable immunogenicity ... Apr 3, 2023 · The company has also pointed to signs, based on comparisons to historical controls, that patients who received SRP-9001 might be faring better than they would without treatment. The FDA has already approved multiple Duchenne drugs, including three of Sarepta’s, based on the idea that boosting dystrophin will lead to better function and health. Oct 4, 2022 · SRP-9001 is designed to deliver a gene encoding micro-dystrophin (a shortened but functional version of the dystrophin protein) to the body’s muscle cells using a viral vector. The therapy is being co-developed by Sarepta and Roche. “If approved, SRP-9001 will be the first gene therapy available for Duchenne patients,” Ingram said. Mar 18, 2023 · In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting. 1 The current Prescription Drug User Fee Act action date for the treatment’s biologics license ... Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. Sarepta’s gene therapy engine is tailored to specific disease states and aims to select individual components that target specific tissues and cells with the goal of optimizing expression of the select protein in those tissues. Together, these three components form what is called a construct . Oct 1, 2022 · Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein). Jan 7, 2021 · Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks ... Mar 18, 2023 · An additional integrated analysis of 52 patients across studies SRP-9001-101, SRP-9001-102, and ENDEAVOR also showed that at 1 year, patients treated with SRP-9001 at the target dose improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (P <.0001). 2. REFERENCES 1 ... Jul 6, 2022 · SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. Data are being presented this week at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in Brussels. Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... Aug 23, 2023 · ³ Other LGMD targets in development: SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), and SRP-9010 (LGMD2A/R1) Download PDF of Our Pipeline For more information about our pipeline, please contact Sarepta's Patient Affairs team at [email protected] . Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene, to muscle tissue, needed for muscle membrane stability. Jul 22, 2022 · Currently, four notable gene therapy candidates are under development: PF-06939926 from Pfizer, SRP-9001 from Sarepta Therapeutics, SGT-1001 from Solid Biosciences, and GNT 0004 from Genethon. There are two Ph3 trials registered at ClinicalTrials.gov to evaluate PF-06939926 and SRP-9001. These trials have an enrollment size of 99–120, and the ... Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. May 12, 2023 · SRP-9001 is supported by non-clinical evidence in addition to efficacy and safety data from studies 101, 102 and 103 as well as an integrated analysis across these three clinical studies comparing ... May 12, 2023 · The vote, 8 to 6, came after a day of testimony from speakers for Sarepta Therapeutics, the maker of the gene therapy called SRP-9001, FDA scientists and families whose children have Duchenne ... We would like to show you a description here but the site won’t allow us. Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene, to muscle tissue, needed for muscle membrane stability. Aug 2, 2022 · Equally impressive SRP-9001 treated patients improved 3.8 points on unadjusted means and 3.2 requiring of 52 weeks on NSAA compared to the propensity Master external control group, with a p-value ... Sep 22, 2022 · Sarepta has already dosed 80 patients in clinical trials for SRP-9001, ranging from 3 to 19 years of age, with varying levels of ambulation. Its phase 3 trial, known as EMBARK, is enrolling 120 patients in the US and overseas. Later this year, Sarepta hopes to launch its second phase 3 study, ENVISION, for boys older than 8 years of age—both ... Jun 22, 2023 · Under this approval pathway, the FDA has granted conditional approval to SRP-9001 (ELEVIDYS), with the requirement that Sarepta complete its ongoing Phase 3 trial (EMBARK) as a confirmatory study. All patients in the confirmatory trial are expected to be treated by September 2023, and a full readout is expected in early 2024. Jan 11, 2022 · Participants in the trial will be given a single infusion of the gene therapy or placebo and be monitored for 52 weeks (about a year). Then, participants originally given SRP-9001 will get an infusion of placebo, and vice versa, followed by another year of monitoring. The study’s main goal is to measure changes in NSAA scores. Apr 3, 2023 · The company has also pointed to signs, based on comparisons to historical controls, that patients who received SRP-9001 might be faring better than they would without treatment. The FDA has already approved multiple Duchenne drugs, including three of Sarepta’s, based on the idea that boosting dystrophin will lead to better function and health. Jun 22, 2023 · SRP-9001 is a novel DMD gene-replacement therapy co-invented by Dr. Mendell and Louise Rodino-Klapac, PhD, formerly at Nationwide Children’s and currently the executive vice president, head of Research and Development, and chief scientific officer at Sarepta Therapeutics. May 12, 2023 · SRP-9001 dystrophin protein expression is reasonably likely to predict clinical benefit (product design, biological, and empirical data showing direct change in underlying pathophysiology of... Apr 22, 2021 · SRP-9001 is an adeno-associated virus (AAV) mediated gene therapy that delivers a micro-dystrophin-encoding gene to the muscles and is intended as a curative treatment. The specific vector deployed in the gene transfer, AAVrh74, has been shown to achieve the efficient delivery of micro-dystrophin to skeletal muscle with tolerable immunogenicity ... May 12, 2023 · SRP-9001 dystrophin protein expression is reasonably likely to predict clinical benefit (product design, biological, and empirical data showing direct change in underlying pathophysiology of... Oct 4, 2022 · SRP-9001 is designed to deliver a gene encoding micro-dystrophin (a shortened but functional version of the dystrophin protein) to the body’s muscle cells using a viral vector. The therapy is being co-developed by Sarepta and Roche. “If approved, SRP-9001 will be the first gene therapy available for Duchenne patients,” Ingram said. A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Jan 11, 2022 · The double-blind, placebo-controlled Study 102 is evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 patients with DMD. Part 2 of the study evaluated SRP-9001 in ... Nov 28, 2022 · The BLA is supported by efficacy and safety data from the SRP-9001-101, SRP-9001-102, and SRP-9001-103 studies (ClinicalTrials.gov Identifier: NCT03375164, NCT03769116, NCT04626674, respectively ... Oct 1, 2022 · Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein). Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. The company noted that SRP-9001 would be the first gene therapy for Duchenne. Sarepta is ... Mar 18, 2023 · In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting. 1 The current Prescription Drug User Fee Act action date for the treatment’s biologics license ... May 3, 2022 · Patients treated with SRP-9001 generally maintained muscle strength (Time to Rise and 4-Stair Climb) and showed improvement in ambulation ability (100m) from baseline to Year 3. Conclusions: The observed safety profile and the enduring response following gene transfer provides proof-of-concept for the continuation of clinical trials assessing ... Apr 10, 2023 · SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of functional components of dystrophin. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving ... Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... We would like to show you a description here but the site won’t allow us. Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... May 12, 2023 · SRP-9001, otherwise known as delandistrogene moxeparvovec, is designed to deliver a copy of a gene encoding microdystrophin to muscle cells using a lab-engineered, harmless virus as a vector. It is prepared as a suspension of concentration of 1.33 x 1013 vg/mL, and is supplied in a single-use, 10 mL vial for a single-dose, intravenous infusion. Jun 22, 2023 · Under this approval pathway, the FDA has granted conditional approval to SRP-9001 (ELEVIDYS), with the requirement that Sarepta complete its ongoing Phase 3 trial (EMBARK) as a confirmatory study. All patients in the confirmatory trial are expected to be treated by September 2023, and a full readout is expected in early 2024. Jul 22, 2022 · Currently, four notable gene therapy candidates are under development: PF-06939926 from Pfizer, SRP-9001 from Sarepta Therapeutics, SGT-1001 from Solid Biosciences, and GNT 0004 from Genethon. There are two Ph3 trials registered at ClinicalTrials.gov to evaluate PF-06939926 and SRP-9001. These trials have an enrollment size of 99–120, and the ... Aug 2, 2022 · Equally impressive SRP-9001 treated patients improved 3.8 points on unadjusted means and 3.2 requiring of 52 weeks on NSAA compared to the propensity Master external control group, with a p-value ... We would like to show you a description here but the site won’t allow us. Jan 7, 2021 · About SRP-9001-102 Study SRPJul 24, 2020 · About SRP-9001 SRP-9001 is an investigatio Jun 22, 2020 · Study 101 was an open-label trial that infused SRP-9001 at a dose of 2x10 14 vg/kg via peripheral arm vein in all 4 of the ambulatory patients with DMD between the ages of 4 and 7 without preexisting AAVrh74 antibodies and a stable corticosteroid dose of ≥12 weeks. In addition to receiving SRP-9001, patients were given daily prednisolone, 1 ... Nov 28, 2022 · SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well ... Jan 11, 2022 · Participants in the trial will be given a May 12, 2023 · In documents released Wednesday, FDA reviewers concluded Sarepta’s clinical trials “do not provide unambiguous evidence” that the therapy, known as SRP-9001, is “likely beneficial for ... May 12, 2023 · SRP-9001 uses viruses to ferry a cop...

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