PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.

NCT Number	NCT07429240
Lead Sponsor	Precision BioSciences, Inc.
Conditions	Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Enrollment	18 participants
Start Date	2026-04-24
Primary Completion	2029-11 (estimated)
Study Completion	2029-12 (estimated)
Updated on ClinicalTrials.gov	2026-06-23