Editas Medicine Reports Promising Preclinical Data for LDL-Cholesterol-Lowering Gene Editing Therapy EDIT-401 at ESGCT Congress

Editas Medicine presented preclinical data for EDIT-401, a gene therapy showing over 90% reduction in LDL cholesterol.

Quiver AI Summary

Editas Medicine, Inc. announced promising preclinical data for its experimental gene editing therapy, EDIT-401, designed to significantly reduce LDL cholesterol levels. Presented at the 32nd Annual European Society of Gene and Cell Therapy Congress in Seville, the data demonstrated over 90% reduction in LDL-C levels in non-human primates after just one dose, with similar results seen in mice. The therapy employs a CRISPR/Cas9 approach to enhance LDL receptor expression, resulting in durable cholesterol reduction maintained over three months in mouse models. Dr. Linda C. Burkly expressed confidence in EDIT-401's potential to improve treatment for individuals with high LDL cholesterol. The oral presentation details were shared, along with information about Editas Medicine's broader commitment to developing transformative gene editing therapies.

Potential Positives

Successful presentation of preclinical proof-of-concept data at a prestigious international congress, highlighting the company's innovative approach to gene editing.
Demonstrated robust efficacy of EDIT-401 with over 90% reduction in LDL-cholesterol within 48 hours, indicating potential for a significant therapeutic impact.
Utilization of advanced CRISPR/Cas9 technology and optimized delivery methods, showcasing Editas Medicine's commitment to leading-edge gene therapy solutions.
Potential for durable LDL-C reduction supported by three-month study results, suggesting long-term benefits for patients dealing with high LDL cholesterol.

Potential Negatives

Dependence on preclinical data: The press release highlights achievements in preclinical studies but does not provide clinical data or effects in human subjects, which may raise concerns about the translational impact of the research.
Potential delays in development: As the product is still in preclinical stages, there may be significant time before EDIT-401 becomes available for patient use, impacting investor confidence and market expectations.
Market competition: The mention of "potential best-in-class" therapy suggests a competitive landscape, raising concerns over whether EDIT-401 can maintain a competitive edge against existing and emerging therapies for LDL-C reduction.

FAQ

What is EDIT-401 and its purpose?

EDIT-401 is an experimental gene therapy aimed at significantly reducing LDL cholesterol levels through gene editing techniques.

What were the key findings presented at the ESGCT Congress?

The findings showed robust efficacy, with ≥90% LDL cholesterol reduction in non-human primates and strong upregulation of the LDL receptor.

How does EDIT-401 work to lower LDL cholesterol?

EDIT-401 uses CRISPR/Cas9 technology to upregulate the LDL receptor, resulting in increased mRNA stability and significant LDL cholesterol reductions.

What are the implications of the preclinical data for patients?

The data suggest EDIT-401 could provide a novel and effective option for individuals with high LDL cholesterol, potentially improving their health outcomes.

Where can I find more information about Editas Medicine and EDIT-401?

More information, including scientific presentations and details about ongoing research, is available on Editas Medicine's official website.

Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.

$EDIT Insider Trading Activity

$EDIT insiders have traded $EDIT stock on the open market 8 times in the past 6 months. Of those trades, 0 have been purchases and 8 have been sales.

Here’s a breakdown of recent trading of $EDIT stock by insiders over the last 6 months:

GILMORE NEIL O'NEILL (CEO) has made 0 purchases and 2 sales selling 20,784 shares for an estimated $40,712.
LINDA BURKLY (EVP, CHIEF SCIENTIFIC OFFICER) has made 0 purchases and 3 sales selling 6,557 shares for an estimated $15,851.
AMY PARISON (SVP, Chief Financial Officer) has made 0 purchases and 3 sales selling 1,583 shares for an estimated $3,709.

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UBS AM, A DISTINCT BUSINESS UNIT OF UBS ASSET MANAGEMENT AMERICAS LLC removed 2,600,000 shares (-100.0%) from their portfolio in Q2 2025, for an estimated $5,720,000
MILLENNIUM MANAGEMENT LLC added 1,590,955 shares (+557.3%) to their portfolio in Q2 2025, for an estimated $3,500,101
MORGAN STANLEY removed 1,238,238 shares (-46.6%) from their portfolio in Q2 2025, for an estimated $2,724,123
WOODLINE PARTNERS LP added 846,399 shares (+inf%) to their portfolio in Q2 2025, for an estimated $1,862,077
NUVEEN, LLC added 633,230 shares (+200.0%) to their portfolio in Q2 2025, for an estimated $1,393,106
UBS GROUP AG removed 586,015 shares (-48.2%) from their portfolio in Q2 2025, for an estimated $1,289,233
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$EDIT Analyst Ratings

Wall Street analysts have issued reports on $EDIT in the last several months. We have seen 2 firms issue buy ratings on the stock, and 0 firms issue sell ratings.

Here are some recent analyst ratings:

HC Wainwright & Co. issued a "Buy" rating on 08/15/2025
Baird issued a "Outperform" rating on 05/13/2025

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$EDIT Price Targets

Multiple analysts have issued price targets for $EDIT recently. We have seen 4 analysts offer price targets for $EDIT in the last 6 months, with a median target of $4.5.

Here are some recent targets:

Mitchell S. Kapoor from HC Wainwright & Co. set a target price of $5.0 on 09/05/2025
Yanan Zhu from Wells Fargo set a target price of $4.0 on 09/03/2025
Joel Beatty from Baird set a target price of $6.0 on 08/13/2025

Full Release

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.