Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD Gene Editing Therapy
Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
Precision BioSciences, Inc. (Nasdaq: DTIL) announced that the FDA has granted Fast Track designation for PBGENE-DMD, a novel gene editing therapy for Duchenne muscular dystrophy. The designation follows the company's recent IND clearance and supports PBGENE-DMD's continued momentum towards clinical investigation in boys with DMD.
PBGENE-DMD is a first-in-class gene editing therapy that utilizes a gene excision approach clearly differentiated from existing microdystrophin and exon skipping treatments. The therapy is designed to potentially provide durable functional muscle improvement for DMD patients with mutations in exons 45-55, representing up to 60% of boys with DMD. A single AAV encodes two ARCUS proteins designed to permanently edit a patient's DNA within the dystrophin gene, resulting in a naturally-expressed, near full-length, functional dystrophin protein.
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 production of a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue, diaphragm 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.
The Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients between the age of 2-7 with mutations between exons 45 and 55. The objective of the FUNCTION-DMD study is to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes in patients afflicted with DMD. The clinical trial is registered at www.clinicaltrials.gov under NCT07429240.
Precision BioSciences also announced a Duchenne Muscular Dystrophy Investor Event, with a live question-and-answer session with investors and analysts to follow the formal presentation. A replay of the webinar will be accessible in the Investors section of Precision's website following the event.
Additionally, the company announced a presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference. The poster presentation, titled "PBGENE-DMD gene editing treatment leads to safe and long-term functional improvement in humanized DMD-disease mouse model" (Publication number: 142M), will present preclinical data supporting the therapy's development.