Dyne Therapeutics Announces Orphan Drug And Rare Pediatric Designations For DYNE-251

Dyne Therapeutics, Inc. (DYN) announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy mutations amenable to exon 51 skipping, was granted FDA orphan drug and rare pediatric disease designations. DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial.

Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a rare pediatric disease.

Wildon Farwell, chief medical officer of Dyne, said: "We continue to advance our DELIVER clinical trial and look forward to sharing initial clinical data later this year."

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