Precision BioSciences, Inc, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).

“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. 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.”

The FDA grants Rare Pediatric Disease Designation to therapies targeting serious and life-threatening diseases that affect fewer than 200,000 people in the U.S. and primarily affect individuals under the age of 18. Approximately 15,000 people in the U.S. alone are living with Duchenne muscular dystrophy.

With this designation, 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.

PBGENE-DMD is Precision’s development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near-full length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address more than 60% of the DMD patient population.

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.

Source: precisionbiosciences.com