Gene Therapy Continues to Show Efficacy in Duchenne Muscular Dystrophy
The placebo crossover cohort experienced statistically significant improvements in NSAA score over external control.
SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy from Sarepta Therapeutics, statistically improved motor function in children with
“We are delighted to report positive results for Part 2 of our blinded, placebo-controlled Study 102 in Duchenne, where the 48-week functional benefits of SRP-9001 in patients dosed at cross-over were statistically significant when compared to pre-specified matched external controls. Furthermore, the safety profile of SRP-9001 remains consistent with the wealth of previous clinical data,” Doug Ingram, president and chief executive officer, Sarepta, said in a statement.1
The ongoing, randomized, double-blind, placebo-controlled clinical trial enrolled 41 children with DMD, 21 of whom were in the placebo crossover cohort, to evaluate SRP-9001. The gene transfer therapy is designed to deliver the micro-dystrophingene to targeted muscle tissue.
Participants in the placebo crossover group (n = 20, age range, 5-8 years) scored a mean of 2.0 points higher on North Star Ambulatory Assessment (NSAA) at 48 weeks compared to external control (n = 103; P = .0009). NSAA scores improved 1.3 points from baseline while scores in the external control group decreased 0.7 points from baseline.
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SRP-9001 continued to be well-tolerated in this second part of the study, with no new safety signals in previously treated patients, no treatment-related serious adverse events (AEs), deaths, or study discontinuations. The most common AE was vomiting. Study participants continue to be monitored for safety and long-term efficacy.
“Study 102, Part 2 results add to the totality of evidence for SRP-9001 generated thus far ‒ with promising results across multiple clinical trials and more than 80 patients dosed, encompassing a wide range of phenotypes as well as the oldest and heaviest Duchenne patients to be dosed with a full body AAV gene therapy infusion to date. The totality of results that we have seen across our multiple trials bolsters our confidence in the potential disease-modifying benefits of this therapy and reinforces our conviction in the probability of success of EMBARK, our large, phase 3 placebo-controlled global study presently underway and dosing. We are reminded daily that Duchenne is a brutal, life-ending disease and SRP-9001 is the greatest near-term hope we all have to address the need for a therapy that changes the trajectory of this disease. We will continue to move as quickly as possible to bring SRP-9001 to patients in the United States and around the world,” Ingram added to the statement.1
Sarepta announced
"With 77 patients treated to date, the multi-study development program for SRP-9001 represents the most comprehensive and long-term dataset for a Duchenne muscular dystrophy gene therapy in existence. The totality of evidence shows that SRP-9001 is a significantly differentiated gene therapy product candidate with one-time dosing and a stable tolerability profile, results in robust expression and evidence of sustained functional benefits across our various studies," Ingram said in an earlier statement.2
SRP-9001 will be further evaluated in the phase 3, double-blind, EMBARK study (Study SRP-9001-301), which will be conducted across the US, Europe, and Asia.3 The study plans to enroll 120 participants with DMD between the ages of 4 to 7. Participants with mutations inclusively between exons 1-17 or mutations in exon 45 are not eligible.
REFERENCES
1. Sarepta Therapeutics’ gene therapy SRP-9001 shows statistically significant functional improvements compared to pre-specified matched external control in part 2 of study SRP-9001-102 for the treatment of Duchenne muscular dystrophy. News release. Sarepta Therapeutics. January 10, 2022. https://finance.yahoo.com/news/sarepta-therapeutics-gene-therapy-srp-155000189.html?guccounter=1
2. Sarepta Therapeutics’ SRP-9001 shows sustained functional improvements in multiple studies of patients with Duchenne. News release. Sarepta Therapeutics. October 11, 2021. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-srp-9001-shows-sustained-functional
3. Sarepta Therapeutics announces initiation of EMBARK, a global pivotal study of SRP-9001, a gene therapy for the treatment of Duchenne Muscular Dystrophy. News release. Sarepta Therapeutics. October 4, 2021. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-initiation-embark-global-pivotal
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