Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress

Rhea-AI Summary

Editas Medicine (NASDAQ: EDIT) reported in vivo preclinical proof-of-concept data for EDIT-401 on October 9, 2025, showing robust LDL-cholesterol lowering in animal models.

Key readouts: ≥90% mean LDL-C reduction in non-human primates within 48 hours and in mice with high baseline LDL-C; a ≥6-fold mean increase in LDLR protein in NHP liver; LDL-C reductions were durable in a three-month mouse study. Data were presented at the ESGCT 32nd Annual Congress and will be posted on the company website.

Positive

• ≥90% mean LDL-C reduction in non-human primates within 48 hours
• ≥90% LDL-C reduction in high-LDL-C mouse models
• ≥6-fold mean increase in LDLR protein in NHP liver
• LDL-C reduction durable in mouse models for three months

Negative

• Data are preclinical only; no human clinical results reported
• Presentation did not disclose safety or tolerability data
• No clinical dosing or human translation timeline provided

Preclinical data demonstrates proof-of-concept for EDIT-401 with ≥90% mean LDL-C reduction in non-human primates and mouse models

CAMBRIDGE, Mass., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today reported in vivo preclinical proof-of-concept data for EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (LDL-C), at the 32^nd Annual European Society of Gene and Cell Therapy (ESGCT) Congress in Seville, Spain. The Company shared results from preclinical studies demonstrating potent and durable reductions in LDL-C through upregulation of the LDL receptor (LDLR).

Key EDIT-401 Data Presented includes:

• Robust efficacy data: ≥90% LDL-C reduction in non-human primates achieved within 48 hours of a single dose of EDIT-401; ≥90% LDL-C reduction in mice with high baseline LDL-C and reduced LDLR function
• Optimized therapeutic strategy:
  • CRISPR/Cas9 nuclease and dual gRNAs with LNP delivery disrupt negative regulatory elements in the 3' UTR, increasing mRNA stability enabling potent LDLR upregulation
  • ≥6-fold mean increase in LDLR protein in the NHP liver, requiring only a moderate level of functional editing of LDLR alleles
• Durable effect: LDL-C reduction maintained in mouse models in a three-month study
  

“The in vivo proof-of-concept data presented today reinforce the potential impact of our differentiated upregulation strategy. In preclinical non-human primate studies, EDIT-401 achieved robust efficacy data with a ≥90% mean LDL-C reduction. These data strengthen our conviction that EDIT-401 represents a novel therapeutic approach with the potential to significantly improve outcomes for people living with high LDL cholesterol,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.

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 at the time of the presentation and will remain accessible following the event.

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

About Editas Medicine 
As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of transformative in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.

Investor and Media Contacts: 
ir@editasmed.com 
media@editasmed.com   

FAQ

What did Editas announce about EDIT-401 at ESGCT on October 9, 2025?

Editas presented preclinical in vivo data showing ≥90% mean LDL-C reduction in non-human primates and mice and a ≥6-fold LDLR protein increase.

How fast did EDIT-401 reduce LDL-C in non-human primates (EDIT)?

EDIT-401 achieved a ≥90% mean LDL-C reduction within 48 hours after a single dose in non-human primates.

Does the Editas (EDIT) announcement include human clinical data for EDIT-401?

No. The announcement reports preclinical animal data only; no human clinical results were disclosed.

What mechanism does EDIT-401 use to upregulate LDLR according to Editas (EDIT)?

EDIT-401 uses a CRISPR/Cas9 nuclease with dual gRNAs and LNP delivery to disrupt 3' UTR regulatory elements, increasing LDLR mRNA stability.

Were durability results reported for EDIT-401 in the Editas (EDIT) presentation?

Yes. LDL-C reduction was reported as maintained in mouse models over a three-month study.

Where and when can investors view the EDIT-401 presentation from Editas (EDIT)?

The presentation was delivered at ESGCT on October 9, 2025 and will be posted in the company’s "Posters & Presentations" section on its website.