Srp 9001 - 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 .

 
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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 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. Nov 30, 2022 · According to a recent announcement, the FDA accepted and granted priority review to Sarepta Therapeutics ’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023. 1. "We are delighted to announce that the FDA has accepted Sarepta ... 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 ... 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 ... 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. 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 ... 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. 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 ... Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... 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. 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 ... 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. Apr 27, 2020 · A detailed picture of the SRP-9001 in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2020-2030 is provided ... 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. 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 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 ... 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 ... 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] . Jul 24, 2020 · About SRP-9001 SRP-9001 is an investigational gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. 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 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. Dec 23, 2019 · SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. 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 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 ... 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 ... Jan 20, 2023 · If Sarepta receives regulatory approval for its SRP-9001 therapy, its combined DMD-related sales could jump to $3.2 billion in DMD-related sales in 2028, led by SRP-9001’s $2 billion contribution. 29 Sarepta expects total peak sales for SRP-9001 to reach $4 billion. 30. SRP-9001 Changing Treatment Landscape for DMD Jul 6, 2022 · In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=<0.0001) 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 · 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 ... 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. 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 ... 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. 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 ... 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. 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 ... 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. 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 · 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 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. 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 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. 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 ... Apr 27, 2020 · A detailed picture of the SRP-9001 in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2020-2030 is provided ... Apr 27, 2020 · A detailed picture of the SRP-9001 in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2020-2030 is provided ... 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. Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... 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. 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. Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... 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 ... 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 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 ... Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... 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. 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 ... 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 ... 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 ... 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 ... 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 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 ... 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 ... 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. We would like to show you a description here but the site won’t allow us. Nov 30, 2022 · According to a recent announcement, the FDA accepted and granted priority review to Sarepta Therapeutics ’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023. 1. "We are delighted to announce that the FDA has accepted Sarepta ... 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 ... 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 ... 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. 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 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 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 ... 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 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 ... 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 ... 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 ... 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 ... Nov 30, 2022 · According to a recent announcement, the FDA accepted and granted priority review to Sarepta Therapeutics ’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023. 1. "We are delighted to announce that the FDA has accepted Sarepta ... We would like to show you a description here but the site won’t allow us. Apr 27, 2020 · A detailed picture of the SRP-9001 in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2020-2030 is provided ...

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 ... . Mcdougal

srp 9001

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 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. 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 Jul 6, 2022 · In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=<0.0001) Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... 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. 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 ... Dec 23, 2019 · SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. Jul 24, 2020 · About SRP-9001 SRP-9001 is an investigational gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. 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 Jul 6, 2022 · In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=<0.0001) Jul 24, 2020 · About SRP-9001 SRP-9001 is an investigational gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Jul 24, 2020 · About SRP-9001 SRP-9001 is an investigational gene transfer therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. 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 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 ... 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. 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. .

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