CAMBRIDGE, Mass. — October 9, 2025 — Leads & Copy — Editas Medicine, Inc. (Nasdaq: EDIT) presented in vivo preclinical proof-of-concept data for EDIT-401, an experimental one-time therapy to reduce LDL-cholesterol (LDL-C), at the 32nd Annual European Society of Gene and Cell Therapy (ESGCT) Congress in Seville, Spain. Preclinical studies demonstrated potent reductions in LDL-C through upregulation of the LDL receptor (LDLR).
Key EDIT-401 data includes ≥90% LDL-C reduction in non-human primates within 48 hours of a single dose, and in mice with high baseline LDL-C and reduced LDLR function. The CRISPR/Cas9 nuclease and dual gRNAs with LNP delivery disrupt negative regulatory elements in the 3′ UTR, increasing mRNA stability, enabling potent LDLR upregulation. Studies showed a ≥6-fold mean increase in LDLR protein in the NHP liver, requiring a moderate level of functional editing of LDLR alleles, and maintained LDL-C reduction in mouse models in a three-month study.
Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine, said the data reinforces the potential impact of their differentiated upregulation strategy, strengthening their conviction that EDIT-401 represents a novel therapeutic approach.
Abstracts are available to registrants on the ESGCT website. The presentation will also be posted to the “Posters & Presentations” section of the Company’s website.
Oral Presentation Details:
Title: A transformative LDL cholesterol-lowering in vivo CRISPR gene editing medicine that functionally upregulates LDLR in mice and non-human primates
Session Date and Time: Thursday, October 9, 5:00 p.m. CEST / 11:00 a.m. ET
Session Title: 9A: Gene Editing II, Ex Vivo Applications
Room: Parallel A
Presenter: Linda Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine
Final Abstract Number: OR069
Contact: ir@editasmed.com, media@editasmed.com
Source: Editas Medicine
