Precision BioSciences Announces Plans for First-In-Human Clinical Trials of PBGENE-DMD Gene Editing Therapy for Duchenne Muscular Dystrophy in 2025

Reuters

07-16

Precision BioSciences Announces Plans for First-In-Human Clinical Trials of PBGENE-DMD Gene Editing Therapy for Duchenne Muscular Dystrophy in 2025

Precision BioSciences, Inc. has announced plans to advance its PBGENE-DMD program, an innovative in vivo gene editing approach for treating Duchenne muscular dystrophy (DMD), towards clinical trials. The company is targeting the submission of an Investigational New Drug $(IND.AU)$ and/or Clinical Trial Application $(CTA.UK)$ by 2025, with initial clinical data expected in 2026. This approach aims to address mutations in the 'hot spot' region between exons 45-55, potentially benefiting up to 60% of DMD patients. Recent preclinical data indicate a significant increase in dystrophin-positive muscle cells, which may translate into improved muscle function. Precision BioSciences plans to present the complete dataset at a future scientific conference.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Precision BioSciences Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20250716326036) on July 16, 2025, and is solely responsible for the information contained therein.

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