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Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD 

BGENE-DMD is designed to treat Duchenne muscular dystrophy (DMD) via a one-time administration in a single AAV.

By: Rachel Klemovitch

Assistant Editor

Precision BioSciences, Inc., a clinical-stage gene editing company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD). The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.  Orphan Drug Designation provides sponsors certain benefits, in...

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