Dyne Therapeutics today announced positive initial clinical data from its DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. Dyne-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to skeletal muscle.

According to Dyne, treatment with DYNE-251 in the 5 mg/kg cohort (4 individuals) surpassed the level of dystrophin production reported for the approved standard of care exon 51 skipping for Duchenne. Additionally, the Phase 1/2 study of DYNE-251 demonstrated favorable safety included through the 20 mg/kg cohort (37 individuals). Dyne indicated that enrollment is complete through the 20 mg/kg cohort of the DELIVER trial (40 total patients enrolled) and approximately 275 doses have been administered to date, supporting dose escalation up to 40 mg/kg.

The company anticipates providing its next clinical data update from the DELIVER trial in the second half of 2024.

Read Dyne’s press release here.