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Dyne Therapeutics’ DYNE-251 Receives the US FDA’s Fast Track Designation for the Treatment of Duchenne Muscular Dystrophy

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Dyne Therapeutics’ DYNE-251 Receives the US FDA’s Fast Track Designation for the Treatment of Duchenne Muscular Dystrophy

Shots:

  • 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

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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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