FDA Grants Fast Track Designation to Precision BioSciences' DMD Gene Therapy
Precision BioSciences receives FDA Fast Track designation for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy designed to treat patients with mutations in exons 45-55.
Precision BioSciences Inc. (NASDAQ:DTIL) announced that the U.S. Food and Drug Administration has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy. Shares rose 13% Monday following the announcement. The clinical-stage biotech company, with a market capitalization of $111 million, has seen its stock rise 11% year-to-date to $4.61, though shares remain 48% below their 52-week high.
The designation is intended to facilitate development and expedite review of drugs for serious conditions with unmet medical need. The Fast Track designation allows for more efficient interactions with the FDA as development progresses. Precision BioSciences received IND clearance for PBGENE-DMD prior to the Fast Track designation.
PBGENE-DMD uses a gene excision approach and is designed for DMD patients with mutations in exons 45-55, which represents up to 60% of boys with the condition. The therapy uses two ARCUS proteins to edit DNA within the dystrophin gene, aiming to produce a near full-length functional dystrophin protein. Precision BioSciences utilizes its proprietary ARCUS platform to develop in vivo gene editing therapies.
The company plans to host a virtual event on March 17, 2026, at 9:00 AM ET featuring a Pediatric Neurologist and Associate Professor of Pediatrics at Arkansas Children's Hospital, and the Founding President of Parent Project Muscular Dystrophy. The presenters will discuss PBGENE-DMD and the planned Phase 1/2 FUNCTION-DMD clinical study.
The FUNCTION-DMD study is expected to enroll ambulatory DMD patients between ages 2-7 with mutations between exons 45 and 55. The trial will evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes.
In preclinical studies, PBGENE-DMD demonstrated the ability to target muscle types involved in DMD progression and produced functional improvements in a humanized DMD mouse model, restoring the body's ability to produce dystrophin protein across multiple muscles including cardiac tissue, diaphragm and skeletal muscle groups.
Precision BioSciences holds more cash than debt on its balance sheet, though the company is quickly burning through cash—typical for clinical-stage biotechs advancing therapies through trials. The company has reported unaudited cash, cash equivalents, and restricted cash totaling approximately $137 million as of December 31, 2025. This financial position is expected to support data milestones through 2028.
The Durham, North Carolina-based company is actively progressing with its Phase 1/2a ELIMINATE-B trial for chronic hepatitis B, involving multiple dosing cohorts. Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting boys.