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Investing.com -- Precision BioSciences (NASDAQ:DTIL) stock rose 5% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its PBGENE-DMD treatment for Duchenne muscular dystrophy (DMD).
The clinical stage gene editing company, which utilizes its proprietary ARCUS platform, received the designation for its potential treatment of DMD, a rare genetic disorder affecting fewer than 200,000 people in the United States.
This regulatory milestone follows the company’s recent receipt of Rare Pediatric Disease designation for the same therapy. The Orphan Drug Designation provides Precision BioSciences with financial incentives to support clinical development and the potential for up to seven years of market exclusivity if the treatment is ultimately approved.
"Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy," said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences.
The company stated it remains in active dialogue with the FDA as it advances PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026.
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