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Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy
- Company Anticipates Reporting Data from Global, Multiple Ascending Dose DELIVER Clinical Trial in the Second Half of 2023 - WALTHAM, Mass., Oct. 31, 2022
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Company Anticipates Reporting Data from Global, Multiple Ascending Dose DELIVER Clinical Trial in the Second Half of 2023 - WALTHAM, Mass., Oct. 31, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial. “Every day is critical for patients and their families, and we are pleased that the FDA granted Fast Track designation as this provides an opportunity to expedite the development of DYNE-251,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “Duchenne is a fatal disease and available therapies offer limited benefit. We are focused on driving toward meaningful clinical data in our DELIVER trial anticipated in the second half of 2023 and continuing to work closely with the DMD community and the FDA to advance a potentially transformative therapy.” The FDA grants Fast Track designation to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, with the ultimate goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval, which may lead to earlier drug approval and access by patients. A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met. About DYNE-251 DYNE-251 is Dyne’s product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, wi...