Editas Medicine shared in vivo proof of concept data on May 13, 2025, for an undisclosed liver target at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The data supports the development of a potentially first-in-class treatment for this indication.
The Company employed an in vivo editing strategy using lipid nanoparticles (LNPs) with CRISPR/Cas RNA cargo for the liver target gene. This approach mimics a naturally occurring, protective variant, leading to the upregulation of the target gene.
The preclinical studies resulted in a meaningful reduction in a clinically relevant disease-specific biomarker in mice. This confirms Editas Medicine's ability to achieve maximal target gene editing within hepatocytes and clinically meaningful biomarker reduction.
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