WALTHAM, Mass., March 16, 2025 — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company developing life-transforming therapies for genetically driven neuromuscular diseases, today announced positive long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251. The trial demonstrated unprecedented and sustained functional improvement at the selected registrational dose of 20 mg/kg Q4W (approximate PMO dose) in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Updated results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
“DYNE-251 offers the potential for a durable and redosable therapy with clinically meaningful and sustained functional improvement in DMD,” said John Cox, President and CEO of Dyne. “The consistency of these data across multiple endpoints underscores DYNE-251’s potential to address the significant unmet need in Duchenne. We are rapidly advancing DYNE-251 toward a readout later this year, with a potential BLA submission for U.S. accelerated approval in early 2026, leveraging dystrophin expression as a surrogate endpoint. If approved, we anticipate rapid adoption by physicians and patients.”
Pat Furlong, Founder and President of Parent Project Muscular Dystrophy, commented, “These long-term data for DYNE-251 in the exon 51 skip amenable population are very encouraging, demonstrating the promise of sustained functional improvement. This exemplifies how the accelerated approval pathway can expedite new therapies for urgent medical needs.”
Kevin Flanigan M.D., Director, Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital, stated, “DYNE-251 has induced near-full length dystrophin expression not previously seen with exon 51 skipping agents, correlating with evidence of clinical efficacy. I look forward to presenting new functional and safety data at the 2025 MDA Clinical & Scientific Conference.”
The DELIVER trial’s updated assessment includes 12-month functional data from 6 patients in the 20 mg/kg Q4W cohort and 18-month functional data from 6 patients in the 10 mg/kg Q4W cohort (transitioned to 20 mg/kg Q4W after month 6). Safety data, as of February 7, 2025, continue to show a favorable profile.
Key Findings:
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- Function: Sustained improvements in multiple functional endpoints (SV95C, NSAA, 10-MWR, Time to Rise from Floor) in both 20 mg/kg and 10 mg/kg Q4W cohorts.
- Dystrophin Expression: Mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content) at 6 months with 20 mg/kg Q4W.
- Safety: Favorable safety profile with no new treatment-related serious adverse events.
Key Milestones:
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- Registrational Expansion Cohort fully enrolled (32 patients), data expected late 2025.
- Potential BLA submission for U.S. accelerated approval in early 2026.
About DYNE-251
DYNE-251 is an investigational therapeutic for DMD amenable to exon 51 skipping, designed for targeted muscle tissue delivery and near-full length dystrophin protein production.
About DMD
DMD is a rare genetic disease causing progressive muscle weakness and loss of function, primarily affecting males.
About Dyne Therapeutics
Dyne Therapeutics develops life-transforming therapies for genetically driven neuromuscular diseases, utilizing its FORCE™ platform for targeted delivery to muscle and the CNS.
About PackGene
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