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.
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