간단한 설명
Editas was one of the first CRISPR companies and once had several cell-therapy programmes. It closed them and rebuilt around a single in vivo candidate: EDIT-401, aimed at the LDL receptor gene in the liver. In monkeys it lowered several different harmful cholesterol-carrying particles by roughly ninety per cent or more, and a first-in-human trial was being prepared.
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EDIT-401 is an in vivo editing candidate targeting LDLR for heterozygous familial hypercholesterolemia. Editas reported approximately 90 per cent or greater mean reductions in LDL-C, Lp(a) and ApoB in non-human primates, with a clinical trial notification submitted and first-in-human dosing planned, early human proof-of-concept expected around the end of 2026 and Part 1 topline data in 2027.
Sources
- Editas Medicine · 2026
Editas Medicine reports new preclinical data demonstrating progress of EDIT-401 ↗