Precision BioSciences, Inc. announced on June 25, 2025, that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) Designation for PBGENE-DMD. This program is being developed for the treatment of Duchenne muscular dystrophy (DMD).
The RPD designation underscores the significant unmet need for new therapeutic options for boys living with DMD, a serious and life-threatening disease affecting fewer than 200,000 people in the U.S. and primarily individuals under 18.
With this designation, Precision BioSciences may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. This voucher can be used to obtain priority review for a different product or can be sold, potentially providing a significant source of non-dilutive capital.
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