Editas Medicine announced on September 2, 2025, the nomination of EDIT-401 as its lead in vivo development candidate. EDIT-401 is an experimental, one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels for the treatment of hyperlipidemia.
EDIT-401 leverages Editas' differentiated upregulation approach, designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression. Preclinical studies in non-human primates demonstrated approximately 90% mean LDL-C reduction with a single dose.
The Company is on track to submit an Investigational New Drug (IND) or Clinical Trial Application (CTA) for EDIT-401 by mid-2026, with the goal of achieving in vivo human proof-of-concept data by the end of 2026. Editas Medicine re-confirmed its cash runway guidance, expecting existing cash to fund operations into the second quarter of 2027.
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