DYNE-251

Dyne Therapeutics reports promising clinical data for DYNE-101 in Myotonic Dystrophy Type 1, planning a registrational expansion cohort for potential U.S. accelerated approval by H1 2026. Also, DYNE-251 shows unprecedented dystrophin expression in Duchenne Muscular Dystrophy, with registrational cohorts expected by end of 2024.

Dyne Therapeutics announced positive clinical data from its Phase 1/2 ACHIEVE trial for DYNE-101 in DM1, showing splicing correction and functional improvements. Plans for a global Registrational Expansion Cohort aim for U.S. Accelerated Approval by H1 2026. DYNE-251 for DMD is also progressing, with data expected late 2025 for potential early 2026 submission.

Dyne Therapeutics received FDA IND clearance for DYNE-101 for DM1, anticipates new ACHIEVE trial data in early 2025, and is enrolling a DELIVER trial cohort for DYNE-251 in DMD. The company reported a Q3 2024 net loss of $97.1M, with $723.7M in cash to fund operations into 2026.

Dyne Therapeutics' DYNE-251, an investigational antisense oligonucleotide, showed significant dystrophin expression in Duchenne muscular dystrophy patients amenable to exon 51 skipping in the DELIVER trial. DYNE-251 achieved a mean absolute dystrophin expression of 3.71% of normal, surpassing the standard of care. The therapy demonstrated functional improvements and a favorable safety profile, prompting the initiation of registrational cohorts with plans for an update on the path to registration by year-end 2024.

Treatment with DYNE-251, Dyne Therapeutics' exon 51-skipping therapy, improved motor function in boys with Duchenne muscular dystrophy (DMD) in a Phase 1/2 trial. DYNE-251 increased dystrophin levels, with 20 mg/kg doses reaching 3.71% of normal, significantly higher than Exondys 51. Functional benefits were observed across various assessments, meeting European Medicines Agency thresholds for clinical significance. Dyne plans to initiate registrational cohorts and pursue expedited approval pathways, with an update expected by year's end.

Dyne Therapeutics announced positive Phase 1/2 DELIVER trial data for DYNE-251 in Duchenne patients, showing 3.71% mean absolute dystrophin expression at 20 mg/kg Q4W, with improvements in functional measures. The company plans to initiate registrational cohorts and pursue expedited approval pathways.

Dyne Therapeutics announces new clinical data from its Phase 1/2 DELIVER trial of DYNE-251 in Duchenne muscular dystrophy, showing unprecedented dystrophin expression and functional improvement. The company plans to initiate registrational cohorts and provide an update on its path to registration by the end of 2024.

Dyne Therapeutics announced significant clinical data from its Phase 1/2 DELIVER trial of DYNE-251 for Duchenne muscular dystrophy, showing unprecedented dystrophin expression and functional improvements. The company plans to initiate registrational cohorts and provide a registration update by end-2024. A virtual investor event was held to discuss these findings.

Dyne Therapeutics' stock fell 31.90% after releasing updated Phase 1/2 DELIVER trial data for DYNE-251 in Duchenne muscular dystrophy patients. DYNE-251 showed higher dystrophin expression levels compared to standard care, with a favorable safety profile. The company plans to update on registration by end of 2024.