Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).

With this, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. The Priority Review Voucher program is designed to incentivize drug development for serious, rare pediatric diseases. 

If awarded, a Priority Review Voucher can be redeemed by the original sponsor to receive FDA priority review for a different product. In addition, the Priority Review Voucher can also be sold to a different sponsor, serving as a source of non-dilutive capital to the original sponsor.

“The receipt of Rare Pediatric Disease Designation highlights the significant unmet need that necessitates new therapeutic options for boys living with DMD,” said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. “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.” 

Approximately 15,000 people in the U.S. alone are living with Duchenne muscular dystrophy.

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

Atwell added, “Our approach is designed to permanently edit a patient’s own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein known to be functional in humans. We look forward to completing the final IND-enabling toxicology studies currently underway and generating initial clinical data which is expected in 2026.”