Editas Medicine Reboots With Cholesterol Gene Therapy (EDIT-401) as Lead Asset

Editas Medicine has selected EDIT-401, a one-time in vivo gene editing therapy for hyperlipidaemia (high cholesterol), as its new lead development asset following a strategic company pivot1 3 5.

EDIT-401 targets the LDLR gene to upregulate LDL receptor expression, aiming to significantly reduce LDL cholesterol (LDL-C) levels where conventional lipid drugs may fall short1 3 5.

Preclinical studies in primates show EDIT-401 achieved around 90% LDL-C reduction with a favorable safety profile3 5.

Editas plans to file for Investigational New Drug (IND) or clinical trial application for EDIT-401 by mid-2026, targeting human proof-of-concept data by the end of 20261 5.

The company restructured in late 2024, shutting down ex vivo programs and cutting R&D costs by 66%, to focus resources on in vivo gene editing and cardiovascular/metabolic disease pipeline1 4 5.

Editas holds substantial cash reserves ($178.5M through Q2 2027), reducing near-term financial risk, and has a scalable platform for metabolic diseases5.

Sources:

1. https://www.cellgenetherapyreview.com/3972-News/621166-Editas-picks-cholesterol-therapy-as-lead-in-vivo-development-candidate/

3. https://www.stocktitan.net/news/EDIT/editas-medicine-nominates-edit-401-an-ldlr-targeted-medicine-as-lead-90ju8bj8jz8t.html

4. https://www.sicklecelldisease.org/2025/01/24/editas-medicine-announces-strategic-transition/

5. https://www.ainvest.com/news/editas-medicine-strategic-pivot-vivo-gene-editing-catalyst-cardiovascular-innovation-valuation-growth-2509/

Sources:

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