Durham, N.C. — Precision BioSciences has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PBGENE-DMD, its investigational gene editing therapy for Duchenne muscular dystrophy (DMD), a severe genetic condition primarily affecting boys.
The designation recognizes the potential of PBGENE-DMD to address a serious and life-threatening pediatric disorder that affects fewer than 200,000 individuals in the U.S. Precision’s therapy, which uses the company’s proprietary ARCUS® platform, is designed to permanently edit the dystrophin gene by removing exons 45–55—a mutation segment that accounts for roughly 60% of DMD cases.
“This designation highlights the urgent need for more effective treatment options for boys living with DMD,” said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. “Our approach aims to restore near full-length, functional dystrophin protein, potentially offering a durable benefit.”
The company is currently completing final preclinical safety studies and expects to begin human trials, with initial clinical data projected for 2026.
The FDA’s Rare Pediatric Disease Designation may also make PBGENE-DMD eligible for a Priority Review Voucher upon approval. Such vouchers are intended to incentivize therapies for rare childhood diseases and can be used to expedite FDA review of another product or sold to other drug developers for funding.
