Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD 

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, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for that use.

PBGENE-DMD was also recently granted FDA Rare Pediatric Disease (RPD) designation for the treatment of DMD.

PBGENE-DMD uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene to restore near full-length dystrophin protein within the body to improve functional outcomes. 

In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. 

PBGENE-DMD restored the body’s ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement.

“Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy,” said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. “Looking ahead, we remain in active dialogue with the FDA as we advance PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026.”