Precision BioSciences’ AAV Gene Editing Therapy PBGENE-DMD Receives FDA Fast Track Designation

March 09, 2026 —

Precision BioSciences announced that the U.S. Food and Drug Administration has granted Fast Track designation to PBGENE-DMD, an investigational AAV gene editing therapy for the treatment of Duchenne muscular dystrophy (DMD). The designation is intended to facilitate development and accelerate regulatory review for therapies addressing serious conditions with significant unmet medical need.

PBGENE-DMD is designed as a single-dose AAV gene editing therapy that uses Precision’s proprietary ARCUS platform to permanently edit the dystrophin gene. The therapy uses an AAV vector to deliver two ARCUS editing proteins, enabling targeted excision of disease-causing mutations and restoring production of a near full-length, functional dystrophin protein. This AAV-delivered gene editing approach is intended to offer a durable treatment option for patients with mutations between exons 45 and 55, which represent up to 60% of boys living with Duchenne muscular dystrophy.

The company recently received Investigational New Drug (IND) clearance for PBGENE-DMD and plans to initiate the Phase 1/2 FUNCTION-DMD clinical trial. The study is expected to enroll ambulatory boys aged 2 to 7 with mutations in the exon 45–55 region of the dystrophin gene. The trial will evaluate the safety, tolerability, and potential efficacy of the AAV gene editing therapy, including dystrophin protein expression and functional outcomes.

Preclinical data supporting PBGENE-DMD demonstrated that the AAV-based gene editing therapy can target multiple muscle types implicated in Duchenne progression. In a humanized mouse model of DMD, treatment restored production of a near full-length dystrophin protein across cardiac tissue, diaphragm, and skeletal muscles. Importantly, the AAV gene editing therapy also edited muscle satellite stem cells, which researchers believe may contribute to long-term durability and sustained functional improvement.

Duchenne muscular dystrophy is a severe, progressive genetic disorder characterized by the absence of functional dystrophin, a protein essential for maintaining muscle integrity. The disease leads to progressive muscle degeneration, loss of ambulation, and life-limiting cardiac and respiratory complications. Investigational AAV gene editing therapies such as PBGENE-DMD aim to address the underlying genetic cause of the disease by restoring dystrophin production through targeted DNA editing.