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Editas Medicine Picks EDIT-401 Gene Editing Drug as Lead In Vivo Development Candidate
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Editas Medicine Picks EDIT-401 Gene Editing Drug as Lead In Vivo Development Candidate
Sep 2, 2025 6:00 AM

08:33 AM EDT, 09/02/2025 (MT Newswires) -- Editas Medicine ( EDIT ) said Tuesday it has nominated EDIT-401, an LDLR-targeted therapy for hyperlipidemia, as its lead in vivo development candidate.

The experimental treatment reduced LDL cholesterol by nearly 90% with a single dose in non-human primates, the company said.

EDIT-401 is designed as a one-time gene editing therapy to increase LDLR protein expression and could offer long-term benefits beyond current lipid-lowering drugs, Editas Medicine ( EDIT ) said.

The company expects human proof-of-concept data by the end of 2026, supported by favorable preclinical safety and efficacy results, it said.

Editas Medicine ( EDIT ) added that it has a strong cash position to finance operations into Q2 2027.

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