Precision BioSciences, Inc. announced on July 23, 2025, that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for PBGENE-DMD. This designation is for the treatment of Duchenne muscular dystrophy (DMD).
Orphan Drug Designation provides sponsors with certain benefits, including financial incentives to support clinical development. It also offers the potential for up to seven years of market exclusivity for the drug in the designated orphan indication in the U.S., if it is ultimately approved for that use.
This regulatory milestone follows the recent receipt of Rare Pediatric Disease designation for PBGENE-DMD. The company is advancing final IND-enabling toxicology studies and remains in active dialogue with the FDA, with initial clinical data anticipated in 2026.
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