Editas Medicine said today its lead candidate EDIT-101, an in vivo CRISPR gene editing treatment for Leber congenital amaurosis-10 (LCA10), showed positive initial clinical data showing it to be safe, and to have generated "signals" of efficacy in two of three patients in the study’s adult mid-dose cohort.
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Editas Medicine said today its lead candidate EDIT-101, an in vivo CRISPR gene editing treatment for Leber congenital amaurosis-10 (LCA10), showed positive initial clinical data showing it to be safe, and to have generated "signals" of efficacy in two of three patients in the study’s adult mid-dose cohort.