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In vivo CRISPR Editing Results in Functional Upregulation of a Liver Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice
CAMBRIDGE, Mass., May 13, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a pioneering gene editing company, today shared in vivo proof of concept data supporting the development of a potentially first-in-class treatment for an undisclosed liver target in a poster presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans. Editas scientists will present the data in a poster session on Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT (6:30 p.m. – 8:00 p.m. ET).
An in vivo editing strategy using lipid nanoparticles (LNPs) with CRISPR/Cas RNA cargo was employed for an undisclosed liver target gene. The strategy mimics a naturally occurring, protective variant, resulting in upregulation of the target gene. This resulted in meaningful reduction in the clinically relevant disease-specific biomarker in mice.
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