The US FDA has granted the FTD to DYNE-251 for DMD mutations amenable to exon 51 skipping. DYNE-251 is being studied in the P-I/II (DELIVER) trial which consists of 24wk. multiple ascending doses, a 24wk. OLE & 96wk. LTE evaluating DYNE-251 in 46 ambulant & non-ambulant males aged 4-16yrs.
The 1EPs are safety, tolerability & change from baseline in dystrophin levels & 2EPs incl. measures of muscle function, exon skipping & PK. The results from the MAD placebo-controlled portion of the trial are expected in H2’23
In preclinical studies with Dyne’s FORCE platform, the therapy showed a robust & durable exon skipping & dystrophin expression in the mdx mouse model in skeletal & cardiac muscle along with a reduction in muscle damage & increased muscle function
Ref: Dyne Therapeutics | Image: Dyne Therapeutics
