PBGENE-DMD is an investigational in vivo gene-editing therapeutic candidate for Duchenne muscular dystrophy being developed by Precision BioSciences. It uses the company’s ARCUS genome-editing platform and is designed to address DMD caused by mutations in the dystrophin gene. PBGENE-DMD delivers two complementary ARCUS nucleases using a single AAV vector to excise exons 45–55 of the dystrophin gene. This targeted excision is intended to restore production of a near full-length dystrophin protein, rather than a synthetic microdystrophin, with the goal of improving muscle structural integrity and function in eligible patients with DMD.
Disease: Duchenne Muscular Dystrophy, DMD, (NCT07429240)
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Candidate summary:
Disease/Disease category:
- Neuromuscular diseases
Official title:
A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)
Phases:
- Phase 1
- Phase 2
Status:
Active recruiting
Country:
- United States
