Precision BioSciences Gets Rare Pediatric Disease Status For Duchenne Muscular Dystrophy Therapy

(RTTNews) - Precision BioSciences, Inc. (DTIL) Wednesday said that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for its gene therapy candidate PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness.

With this designation, Precision may be eligible to receive a Priority Review Voucher if PBGENE-DMD is approved by the FDA. This voucher can be used to shorten the FDA review time for a different product or sold to another company.

Final IND-enabling toxicology studies of PBGENE-DMD are underway with initial data expected in 2026. "Our first-in-class PBGENE-DMD program, which leverages the differentiated capabilities of our ARCUS gene editing platform to excise exons 45-55 of the dystrophin gene, holds the potential to drive meaningful improvement and durable functional benefit over time for up to 60% of patients with DMD," said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences.