Treatment · Familial hypercholesterolemia

EDIT-401

Editas Medicine's lead in vivo candidate after abandoning its cell-therapy programmes — targeting LDLR to lower several atherogenic lipoproteins at once.

Preclinical in vivoLDLR

Preclinical research Tested in cells and animals only. Most preclinical programmes never reach people, and animal results often do not carry over.

Simple explanation

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.

Go deeper

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 ↗

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Key facts

Company	Editas Medicine
Disease	Heterozygous familial hypercholesterolemia
Technology	In vivo CRISPR editing
Target gene	LDLR
Development stage	Preclinical, entering first-in-human
Preclinical result	~90 per cent or greater mean reduction in LDL-C, Lp(a) and ApoB in non-human primates Animal data. Non-human primate results do not reliably predict human outcomes.

Cite this page

The CRISPR Atlas. “EDIT-401.” Updated Aug 25, 2026. https://crisprdecoded.com/treatments/edit-401

Connected in the Atlas

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Technologies

CRISPR-Cas9

Diseases

High Cholesterol and Familial Hypercholesterolemia

Genes

LDLR

Companies

Editas Medicine

Educational information only This page is a reference, not medical advice. Research and regulatory status change; check the last-updated date above and confirm anything important against the primary sources listed.