Clinical stage gene editing company Precision BioSciences (Nasdaq:DTIL) announced on Monday that the US Food and Drug Administration (FDA) has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
This designation is intended to facilitate development and expedite review of therapies for serious conditions with unmet medical need.
PBGENE-DMD is a first-in-class gene editing therapy using a single AAV to deliver two ARCUS proteins designed to permanently edit the dystrophin gene, producing near full-length functional dystrophin. The therapy targets mutations in exons 45-55, representing up to 60% of boys with DMD. Preclinical studies demonstrated durable functional improvements across key muscles, including cardiac, diaphragm, and skeletal tissue, and successfully edited muscle satellite cells critical for long-term efficacy.
Precision BioSciences believes the Fast Track designation underscores both the potential of PBGENE-DMD and the urgent need for new treatment options for DMD.
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