On April 28, 2026 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for high unmet need diseases, reported that new preclinical data from its PBGENE-DMD program have been accepted for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts. The accepted abstract highlights new data demonstrating compelling efficacy observed in early-juvenile mice supporting the potential benefit of earlier intervention with PBGENE-DMD in younger patient populations. These data build on previously shared updates showing treatment with PBGENE-DMD leads to durable functional improvement in a humanized Duchenne muscular dystrophy (DMD) mouse model.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Details of the presentation:
Abstract title: PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model
Session: Emerging molecular therapeutic strategies for muscular dystrophies
Presenter: Adam Michler Ph.D., DMD Research Lead
Presentation Type: Oral Presentation
Presentation Time: May 14, 2026 at 8:45 a.m. ET
About PBGENE-DMD, A Muscle-Targeted Excision Program
PBGENE-DMD is Precision’s development program for the treatment of Duchenne Muscular Dystrophy (DMD), a devastating genetic disease caused by mutations in the dystrophin gene that prevents production of the dystrophin protein, which is essential for maintaining muscle structural integrity and function. DMD affects approximately 15,000 patients in the U.S. alone, and there are currently no approved therapies capable of driving significant, durable functional improvements over time.
PBGENE-DMD is designed to durably improve function for approximately 60% of patients with DMD by employing two complementary ARCUS nucleases, delivered using a single AAV, to excise exons 45-55 of the dystrophin gene, restoring expression of a near full-length dystrophin protein. This protein more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches, which offer minimal functional benefit. Precision’s Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients with mutations between exons 45 and 55, which impact approximately 60% of boys with DMD. The clinical trial will employ an appropriate immune modulation regimen and safety monitoring program to treat patients at world class specialized DMD clinical sites.
PBGENE-DMD was granted Orphan Drug Designation by the FDA in July 2025. The PBGENE-DMD program is eligible for a Priority Review Voucher (PRV) via the Rare Pediatric Disease Priority Review Voucher (PRV) program, which was signed into law on February 3, 2026, as part of the Consolidated Appropriations Act of 2026. PBGENE-DMD received Fast Track designation from the FDA in February 2026.
Further details on the trial can be found on Precision’s website and clinicaltrials.gov identifier NCT07429240.
(Press release, Precision Biosciences, APR 28, 2026, View Source [SID1234664862])