September 30, 2025 — Leads & Copy — Precision BioSciences, Inc. (Nasdaq: DTIL) will present preclinical data for PBGENE-DMD at the 30th Annual International Congress of the World Muscle Society (WMS) in Vienna, Austria, October 7-12, 2025.
According to Dr. Cassie Gorsuch, PhD, Chief Scientific Officer at Precision BioSciences, there remains a significant need for new therapeutic options for patients living with DMD, and PBGENE-DMD holds the potential to be a first-in-class gene editing approach for up to 60% of patients who are impacted by dystrophin mutations between exons 45-55.
The preclinical data demonstrates PBGENE-DMD’s ability to drive significant and sustained improvements in muscle function over time through increased frequency of dystrophin positive cells, increased dystrophin protein expression, and satellite stem cell editing. These encouraging results underscore PBGENE-DMD’s potential to transform the treatment paradigm for patients with DMD, and Precision remains on track to file an IND and/or CTA filing by the end of 2025 with clinical data anticipated in 2026.
The presentation, scheduled for Friday, October 10, 2025, from 3:45-4:45 PM CET, will feature data showing durable improvements in muscle function over time through increased dystrophin expression and dystrophin-positive cells following treatment with PBGENE-DMD.
Precision’s approach is designed to permanently edit a patient’s own DNA sequence, resulting in naturally produced, near full-length dystrophin protein known to be functional in humans. Utilizing two ARCUS nucleases delivered by a single AAV, PBGENE-DMD demonstrated durable improvements in muscle function over time through increased dystrophin expression and dystrophin-positive cells. In a DMD mouse model, PBGENE-DMD was administered at doses up to 1×1014 vg/kg, and mice were evaluated at 3- and 9-months post-dosing for molecular and functional outcomes. Following treatment, dystrophin protein was detected in all muscles evaluated, with increased expression observed at 9 months versus prior timepoints in the quadriceps, gastrocnemius, heart, and diaphragm resulting in substantial and sustained functional muscle improvement. Additionally, an increase in dystrophin-positive muscle cells was observed in all muscles, with up to 85% dystrophin-positive cells in the gastrocnemius. The maximum force output was significantly improved over untreated DMD mice at 3-, 6- and 9-months post-treatment, highlighting strong durability of PBGENE-DMD outcomes. ARCUS-edited dystrophin transcript was also observed in PAX7+ cells, a marker for satellite stem cells, supporting the potential for long-term durability.
Precision continues to advance the final toxicology studies with an anticipated investigational new drug (IND) and/or clinical trial application (CTA) filing targeted by the end of 2025 with initial clinical data expected in 2026.
About PBGENE-DMD:
PBGENE-DMD is Precision’s development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near full-length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address up to 60% of the DMD patient population.
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 the body’s ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue 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.
Contact:
Cassie Gorsuch, Ph.D.
Chief Scientific Officer, Precision BioSciences
Source: Precision BioSciences, Inc.
