Precision BioSciences to present DMD gene therapy data at ASGCT 2026

Precision BioSciences Inc. (NASDAQ: DTIL) will present preclinical data from its PBGENE-DMD gene editing program at the American Society of Gene and Cell Therapy annual meeting in Boston on May 14, 2026.

The oral presentation will highlight data from early-juvenile mice studies supporting potential benefits of earlier intervention with PBGENE-DMD in younger patient populations. The company stated the data builds on previous results showing the treatment leads to functional improvement in a humanized Duchenne muscular dystrophy mouse model.

PBGENE-DMD is designed to treat Duchenne muscular dystrophy, a genetic disease affecting approximately 15,000 patients in the United States. The treatment uses two ARCUS nucleases delivered via a single AAV to excise exons 45-55 of the dystrophin gene, aimed at restoring expression of a near full-length dystrophin protein.

The therapy is designed to address approximately 60% of DMD patients with mutations between exons 45 and 55. The company's Phase 1/2 FUNCTION-DMD study will enroll ambulatory DMD patients with these specific mutations.

PBGENE-DMD received Orphan Drug Designation from the FDA in July 2025 and Fast Track designation in February 2026. The program is eligible for a Priority Review Voucher under the Rare Pediatric Disease Priority Review Voucher program.

The presentation, titled "PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model," will be delivered by Adam Michler, the company's DMD Research Lead, at 8:45 a.m. ET during the muscular dystrophies session.

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