Precision BioSciences (NASDAQ:DTIL) saw its stock rise 5% following the announcement that the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation to its PBGENE-DMD therapy for Duchenne muscular dystrophy (DMD).
The clinical-stage gene editing company, which uses its proprietary ARCUS platform, earned the designation for its potential to treat DMD, a rare genetic condition impacting fewer than 200,000 individuals in the U.S.
This regulatory recognition comes after Precision BioSciences recently received Rare Pediatric Disease designation for the same candidate. The Orphan Drug status offers financial benefits to support clinical development and may grant up to seven years of market exclusivity upon approval.
“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 confirmed ongoing discussions with the FDA as it progresses PBGENE-DMD through regulatory steps, with clinical trial data expected in 2026.
Precision BioSciences stock price
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.