Precision BioSciences, Inc. presented preclinical data for its PBGENE-DMD development program for Duchenne muscular dystrophy (DMD) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference on March 19, 2025.
The data demonstrated significant functional improvement in a humanized DMD mouse model by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. This approach aims to restore the body's native production of a functional dystrophin protein.
Importantly, dystrophin gene correction was observed in muscle satellite stem cells, suggesting potential for long-term durability and functional benefit. This therapeutic approach is applicable for up to 60% of DMD patients, offering broader applicability than current exon skipping approaches.
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