Srp 9001

Jan 10, 2022 · SRP-9001-treated participants from the placebo crossover group (n=20, aged 5-8 at time of dosing SRP-9001) scored a statistically significant 2.0 points higher on the mean North Star Ambulatory ... 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 ... 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 . 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 ... May 12, 2023 · SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Hennick and many other parents like her advocated for the treatment’s accelerated... We would like to show you a description here but the site won’t allow us. Jan 5, 2023 · Ms. Borland discusses SRP-9001’s mechanism of action in terms of its 3 essential components, its vector, promoter, and transgene. She begins by explaining that SRP-9001 uses AAVrh74 as a vector, which was selected for its tissue selectivity for skeletal and cardiac muscle, as well as its low potential for pre-existing antibodies. 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. 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. 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 ... 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... Jun 22, 2023 · For SRP-9001, the surrogate endpoint is a gene called micro-dystrophin, which codes for a shortened form of the dystrophin protein, measured 12 weeks after treatment with SRP-9001. In the US, there are four drugs which already have accelerated access for DMD, three of which are Sarepta’s, and all of which are exon skipping treatments. 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. Sep 29, 2022 · 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 ... Sep 29, 2022 · 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 ... 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 ... 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 ... 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. 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 ... May 18, 2023 · Holly Fernandez Lynch, JD, MBe, is a bioethics and law expert. On May 12, independent experts narrowly recommended that the FDA grant accelerated approval to Sarepta's new gene therapy, SRP-9001 ... May 12, 2023 · SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Hennick and many other parents like her advocated for the treatment’s accelerated... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. 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 ... Jun 22, 2023 · SRP-9001 could become a new standard of care in DMD. Mgmt. has suggested the peak revenue opportunity could exceed $4bn. SRPT share price fell by half when SRP-9001 flunked a Phase 2 study in ... 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 ... Jun 22, 2023 · For SRP-9001, the surrogate endpoint is a gene called micro-dystrophin, which codes for a shortened form of the dystrophin protein, measured 12 weeks after treatment with SRP-9001. In the US, there are four drugs which already have accelerated access for DMD, three of which are Sarepta’s, and all of which are exon skipping treatments. Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... 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 ... 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 ... Jun 22, 2023 · The FDA has approved Sarepta Therapeutics’ investigational gene therapy SRP-9001, otherwise known as delandistrogene moxeparvovec, for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... Nov 28, 2022 · The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Extensive pre-clinical evidence also formed part of the BLA. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. 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. 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 ... We would like to show you a description here but the site won’t allow us. Oct 11, 2021 · In new analyses presented at “SRP-9001 Micro-dystrophin Day,” results from participants treated with SRP-9001 in Study SRP-9001-101 (n=4, ages 4 to 7) found that participants in Study 101 improved 8.6 points on the North Star Ambulatory Assessment (NSAA)* compared to a matched natural history cohort three years following a single ... Aug 2, 2022 · SRP-9001 (delandistrogene moxeparvovec) is designed to deliver a gene encoding micro-dystrophin, a shortened but functional version of this protein, to muscle cells. The therapy delivers its genetic cargo using a specially designed viral vector. Jun 22, 2023 · SRP-9001 could become a new standard of care in DMD. Mgmt. has suggested the peak revenue opportunity could exceed $4bn. SRPT share price fell by half when SRP-9001 flunked a Phase 2 study in ... 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. Nov 29, 2022 · SRP-9001 is designed to deliver a copy of a gene encoding micro-dystrophin — a shortened but functional version of the dystrophin protein, whose lack causes DMD — to muscle cells using a lab-engineered, harmless virus as a vector. 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 ... Nov 28, 2022 · The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Extensive pre-clinical evidence also formed part of the BLA. 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. Jun 22, 2023 · For SRP-9001, the surrogate endpoint is a gene called micro-dystrophin, which codes for a shortened form of the dystrophin protein, measured 12 weeks after treatment with SRP-9001. In the US, there are four drugs which already have accelerated access for DMD, three of which are Sarepta’s, and all of which are exon skipping treatments. 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] . 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 ... Oct 11, 2021 · In new analyses presented at “SRP-9001 Micro-dystrophin Day,” results from participants treated with SRP-9001 in Study SRP-9001-101 (n=4, ages 4 to 7) found that participants in Study 101 improved 8.6 points on the North Star Ambulatory Assessment (NSAA)* compared to a matched natural history cohort three years following a single ... Jun 22, 2023 · For SRP-9001, the surrogate endpoint is a gene called micro-dystrophin, which codes for a shortened form of the dystrophin protein, measured 12 weeks after treatment with SRP-9001. In the US, there are four drugs which already have accelerated access for DMD, three of which are Sarepta’s, and all of which are exon skipping treatments. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. 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 ... Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... 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. 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 ... 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 . 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 ... 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 ... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. Nov 29, 2022 · SRP-9001 is designed to deliver a copy of a gene encoding micro-dystrophin — a shortened but functional version of the dystrophin protein, whose lack causes DMD — to muscle cells using a lab-engineered, harmless virus as a vector. May 11, 2023 · SRP-9001 is designed to deliver to muscle cells a gene encoding micro-dystrophin, a shortened version of the dystrophin protein, which is a protein that is lacking in DMD patients. Sarepta’s application is specifically based on data suggesting the therapy can increase micro-dystrophin levels as designed. Sep 29, 2022 · 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 ... We would like to show you a description here but the site won’t allow us. We would like to show you a description here but the site won’t allow us. 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 ... 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. Jan 4, 2021 · A natural history comparison cohort was drawn retrospectively from the ImagingDMD study. We identified 54 participants who had a total of 110 study visits (between 2011 and 2018) aged between 4.9 and 7.9 years, matching the age of the participants who received SRP-9001. All participants in the natural history cohort were treated with ... 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... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. Jan 5, 2023 · Ms. Borland discusses SRP-9001’s mechanism of action in terms of its 3 essential components, its vector, promoter, and transgene. She begins by explaining that SRP-9001 uses AAVrh74 as a vector, which was selected for its tissue selectivity for skeletal and cardiac muscle, as well as its low potential for pre-existing antibodies. 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 ... 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... May 18, 2023 · Holly Fernandez Lynch, JD, MBe, is a bioethics and law expert. On May 12, independent experts narrowly recommended that the FDA grant accelerated approval to Sarepta's new gene therapy, SRP-9001 ... Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... Jan 4, 2021 · A natural history comparison cohort was drawn retrospectively from the ImagingDMD study. We identified 54 participants who had a total of 110 study visits (between 2011 and 2018) aged between 4.9 and 7.9 years, matching the age of the participants who received SRP-9001. All participants in the natural history cohort were treated with ... 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 . 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. 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. 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 . 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 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 ... Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 We would like to show you a description here but the site won’t allow us. We would like to show you a description here but the site won’t allow us. Aug 2, 2022 · SRP-9001 (delandistrogene moxeparvovec) is designed to deliver a gene encoding micro-dystrophin, a shortened but functional version of this protein, to muscle cells. The therapy delivers its genetic cargo using a specially designed viral vector. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. Jun 22, 2023 · SRP-9001 could become a new standard of care in DMD. Mgmt. has suggested the peak revenue opportunity could exceed $4bn. SRPT share price fell by half when SRP-9001 flunked a Phase 2 study in ... Jun 22, 2023 · SRP-9001 could become a new standard of care in DMD. Mgmt. has suggested the peak revenue opportunity could exceed $4bn. SRPT share price fell by half when SRP-9001 flunked a Phase 2 study in ... Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... 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 ... 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 ... 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 ... Jan 10, 2022 · SRP-9001-treated participants from the placebo crossover group (n=20, aged 5-8 at time of dosing SRP-9001) scored a statistically significant 2.0 points higher on the mean North Star Ambulatory ... 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 ... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. 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 ... Mar 21, 2022 · Four boys with Duchenne muscular dystrophy (DMD) show improved motor function and walking ability three years after being given the experimental gene therapy SRP-9001 in a Phase 1 clinical trial, new data reveal. The findings were presented in a poster, titled “ Phase 1/2A Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with ... 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. May 18, 2021 · About SRP-9001-103 (ENDEAVOR) Study SRP-9001-103 (Study 103) is an open-label clinical trial of SRP-9001 that has enrolled 20 participants with Duchenne muscular dystrophy between the ages of 4-7. 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 5, 2021 · SRP-9001 is one of the leading investigational gene therapies that is being developed for treating DMD and the EMBARK study is the first pivotal study to evaluate any gene therapy for the indication. Jan 10, 2022 · SRP-9001-treated participants from the placebo crossover group (n=20, aged 5-8 at time of dosing SRP-9001) scored a statistically significant 2.0 points higher on the mean North Star Ambulatory ... 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. 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). Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. | qidmkl (article) | qhwhcrz.