Precision BioSciences Wins FDA Orphan Status for Duchenne Gene Therapy

Precision BioSciences said the U.S. Food and Drug Administration has granted Orphan Drug Designation to PBGENE-DMD, its in-vivo gene-editing therapy being developed for Duchenne muscular dystrophy (DMD). The status gives the Durham, North Carolina-based company access to tax credits, user-fee waivers and, if the therapy is ultimately approved, up to seven years of market exclusivity for the rare-disease indication. PBGENE-DMD employs two ARCUS nucleases delivered in a single AAV vector to excise exons 45-55 of the dystrophin gene, an approach the company says could address roughly 60% of DMD patients. The candidate previously received the FDA’s Rare Pediatric Disease designation. Precision is completing investigational new drug-enabling studies and expects first human data in 2026.