EDIT Editas Medicine Inc.

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

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

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 32nd 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 . The presentation will also be posted to the “” 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



Investor and Media Contacts: 
  
    
EN
09/10/2025

Underlying

To request access to management, click here to engage with our
partner Phoenix-IR's CorporateAccessNetwork.com

Reports on Editas Medicine Inc.

 PRESS RELEASE

Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at ...

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 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 32nd An...

 PRESS RELEASE

Editas Medicine Announces EDIT-401 Oral Presentation at the European S...

Editas Medicine Announces EDIT-401 Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress and Participation in Upcoming Investor Conferences CAMBRIDGE, Mass., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced an oral presentation at the upcoming European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 7-10, 2025, in Seville, Spain. European Society of Gene and Cell Therapy (ESGCT) Congr...

 PRESS RELEASE

Editas Medicine Nominates EDIT-401, an LDLR-Targeted Medicine, as Lead...

Editas Medicine Nominates EDIT-401, an LDLR-Targeted Medicine, as Lead In Vivo Development Candidate EDIT-401 achieved ~90% mean LDL-C reduction with single dose in non-human primates EDIT-401 on track for human proof-of-concept data by end of 2026 Strong cash position with operational runway into second quarter of 2027 Company-sponsored webinar on EDIT-401 today at 8:00 a.m. ET CAMBRIDGE, Mass., Sept. 02, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced the nomination of its lead in vivo development candidate, EDIT-401,...

 PRESS RELEASE

Editas Medicine to Host Webinar Announcing Lead In Vivo Development Ca...

Editas Medicine to Host Webinar Announcing Lead In Vivo Development Candidate CAMBRIDGE, Mass., Aug. 28, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced that it will host a webinar on Tuesday, September 2, 2025, at 8:00 a.m. ET to announce the selection of its lead in vivo development candidate. Webinar Presentation Details:The live and archived webcast of the Company’s webinar presentation will be accessible through this , or through the page of the “Inv...

 PRESS RELEASE

Editas Medicine to Participate in Upcoming Investor Conferences

Editas Medicine to Participate in Upcoming Investor Conferences CAMBRIDGE, Mass., Aug. 27, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced that management will participate in the following upcoming investor conferences in September: Wells Fargo Healthcare ConferenceFormat: One-on-one MeetingsDate: Wednesday, September 3Location: Boston, MA Cantor Global Healthcare ConferenceFormat: Fireside Chat with Webcast and One-on-one MeetingsDate: Thursday, Septembe...

ResearchPool Subscriptions

Get the most out of your insights

Get in touch