Editas picks cholesterol therapy as lead in vivo development candidate

Gene editing company Editas Medicine announced the nomination of a lead in vivo development candidate, EDIT-401, a one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels.

EDIT-401 is an in vivo gene editing medicine, based on Editas’ differentiated upregulation approach. EDIT-401 is designed to treat hyperlipidemia (high cholesterol) by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels. This targeted approach has demonstrated a favorable preclinical profile in both efficacy data and tolerability and supports the potential of EDIT-401 to deliver meaningful clinical outcomes for patients underserved by current lipid-lowering therapies.

Last December, Editas unveiled a “strategic transition” to an in vivo gene editing company. The pivot came with a 65% reduction in headcount and an end to the development of reni-cel — the company’s ex vivo autologous treatment targeting sickle cell and transfusion dependent beta thalassemia.

Editas plans 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 for EDIT-401 by the end of 2026. While Editas plans to focus its resources on the advancement of the lead candidate, the company says it remains committed to progressing its pipeline, including optimizing candidates for its hematopoietic stem cell program.