Precision BioSciences Inc., a clinical-stage gene editing company, has received the FDA Rare Pediatric Disease Designation for its PBGENE-DMD therapy aimed at treating Duchenne muscular dystrophy (DMD). This designation recognizes the significant unmet medical need for therapeutic options for individuals with DMD, a severe genetic disorder affecting approximately 15,000 people in the U.S., primarily those under 18 years old. The company's novel ARCUS platform is being used to develop this in vivo gene editing therapy, which targets a specific portion of the dystrophin gene. Precision BioSciences is conducting final IND-enabling toxicology studies, with initial clinical data expected in 2026. The FDA designation may enable Precision to receive a Priority Review Voucher upon PBGENE-DMD's approval, which could expedite the review process for future products or be sold as a financial asset.