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Precision BioSciences Gains FDA Rare Pediatric Disease Designation for PBGENE-DMD

Precision may also be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD.

By: Rachel Klemovitch

Assistant Editor

Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD). With this, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. The Priority Review Voucher program is designed to incentivize drug development for serious, rare pediatric diseases.  If awarded, a Priority Review Voucher can be redeemed by the original sponsor to ...

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