EDIT-401 NHP Preclinical Data Shows 6-Month LDL Lowering, Mid:26 IND On Track

研报英文原文证据摘录

EDIT-401 NHP Preclinical Data Shows 6-Month LDL Lowering, Mid:26 IND On Track

TD Cowen Editas Medicine

Global Research May 14, 2026

In our opinion, '401 has promising preclinical data and addresses a large market in the

hyperlipidemia space. Though statins and PCSK9i's can sufficiently reduce LDL levels for

many people, there remains a need for yet more effective therapies particularly for the

1.2MM people with HeFH and the 10MM with atherosclerotic cardiovascular disease (ASCVD)

who do not achieve LDL-C goals. The history of genomic therapies would suggest that a

convenience advantage is not sufficient for commercial success, but rather increased efficacy

in a population of unmet need combined with good safety will be necessary for adoption. With

90% sustained reductions in LDL-C and benign safety, the NHP data suggest that '401 could

fit this description. Editas believes human POC will be established should '401 demonstrate

acceptable safety with reductions in LDL-C of 60-90%. We look forward to initial clinical data

to further define '401's clinical profile.

The News: Editas Presents New Preclinical Data Further Characterizing

EDIT-401 Including 6 Months Of LDL-C Reductions In NHPs

Editas' lead program, EDIT-401, is a CRISPR/Cas9 therapeutic that aims to edit the 3' UTR

of the LDLR gene, conferring additional mRNA stability and increased protein translation,

and thus decreasing circulating LDL-C levels for the treatment of hyperlipidemia. EDIT-401

leverages GalNAc conjugated LNPs to transiently deliver the gene editing machinery and

gRNAs responsible for installing the intended genetic edit that was originally identified in an

Icelandic family with inherently low levels of LDL-C (74% below population mean) and ~1.5-2.5x

higher LDLR expression.