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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

Key Takeaway: Editas Medicine has reported promising preclinical proof-of-concept data for EDIT-401, a gene therapy aimed at reducing LDL cholesterol. At the ESGCT Congress, the company highlighted that EDIT-401 achieved over 90% mean reduction in LDL-C in non-human primate studies. This innovative approach could greatly benefit individuals with high LDL cholesterol levels.
Price reaction · baseline $4.085 (2025-10-09T15:00:00.000Z) · hit during market hours · clean, no other EDIT news in the window
day 0 close · peak
-0.1%

Market Sentiment Analysis

POSITIVE FACTORS

  • EDIT-401 shows robust efficacy with over 90% mean LDL-C reduction.
  • Potential to significantly improve outcomes for patients with high LDL cholesterol.
  • Presented at a prestigious international congress, enhancing visibility.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+125%
120-day peak, hindsight
Typical move
5.8%
average across 6 past catalysts
Cash runway
~21 mo
Low dilution risk
Lead asset
EDIT-301
Phase 1 · Transfusion Dependent Beta Thalassemia

Full Press Release Details

CAMBRIDGE, Mass., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today reportedin vivopreclinical 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 32ndAnnual 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:

“Thein vivoproof-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 theESGCT 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:
About Editas MedicineAs 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 transformativein vivomedicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize durable, precisionin vivogene 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.

Frequently Asked Questions

What is EDIT-401?

EDIT-401 is an experimental gene therapy aimed at significantly reducing LDL cholesterol.

What were the results of the EDIT-401 studies?

Preclinical studies showed over 90% mean reduction in LDL-C in non-human primates.

Where was the data for EDIT-401 presented?

The data was presented at the 32nd Annual ESGCT Congress in Seville, Spain.

What is the significance of the LDL receptor upregulation?

Upregulation of the LDL receptor is key to achieving durable reductions in LDL cholesterol.

Last updated: Oct 9, 2025