Editas Medicine to Present Preclinical Data Demonstrating Progress in the Development of an in vivo Gene Editing Pipeline at the American Society of Gene and Cell Therapy Annual Meeting

Rhea-AI Summary

Editas Medicine (Nasdaq: EDIT) announced the acceptance of five abstracts for presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), scheduled for May 13-17, 2025, in New Orleans.

The presentations include:

• An oral presentation on in vivo preclinical data using targeted lipid nanoparticles for HBG1/2 promoter editing
• Preclinical proof of concept for a liver target using CRISPR editing
• Results from mouse and non-human primate studies showing high levels of target gene editing in the liver
• Data on guide modification and targeting improvements for better gene editing outcomes

Linda C. Burkly, Executive Vice President and Chief Scientific Officer, highlighted the company's progress in developing in vivo medicines. The research demonstrates their ability to increase protein levels to address diseases caused by loss of function or mutations through gene upregulation editing strategy. Their targeted lipid nanoparticle delivery system shows promise for multiple tissue applications using a 'plug 'n play' approach.

Positive

• Five abstracts accepted for presentation at ASGCT, including one oral presentation, demonstrating research progress
• Successful preclinical proof of concept showing high levels of target gene editing in liver with biomarker response
• Demonstrated capability to increase protein levels to address loss-of-function diseases via gene upregulation strategy
• Progress in targeted lipid nanoparticles (tLNP) delivery system showing potential for multi-tissue applications
• Positive preclinical data from both humanized mouse and non-human primate studies

Negative

• Still in preclinical stage with no immediate revenue generation
• No clinical trial data or timeline announcements provided
• Multiple competitors in the gene editing space could affect market position

CAMBRIDGE, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that five abstracts have been accepted for presentation, including one oral presentation, at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held May 13 – 17, 2025, in New Orleans, LA, and virtually. The Company is presenting preclinical data to support its development of transformative in vivo gene editing medicines.

Editas Medicine presentations at ASGCT include:

• Oral presentation of in vivo preclinical data from humanized mouse and non-human primate (NHP) studies using targeted lipid nanoparticles (tLNPs) to deliver HBG1/2 promoter editing cargo to hematopoietic stem and progenitor cells (HSPCs) and/or hematopoietic stem cells (HSCs) in bone marrow.
• Preclinical proof of concept for an undisclosed liver target using in vivo CRISPR editing to upregulate target protein expression and reduce a disease-associated biomarker in a relevant mouse disease model.
• Proof of concept results from the first in vivo mouse and NHP studies demonstrating high levels of target gene editing in the liver and corresponding biomarker response following intravenous administration of AsCas12a messenger RNA (mRNA) and chemically modified guide RNAs (gRNAs) delivered using LNPs from Genevant.
• Additional preclinical data demonstrating in vivo gene editing capabilities towards developing transformative in vivo medicines, including guide modification and targeting moiety optimizations to increase potency and improve gene editing outcomes in vivo.

“Editas Medicine is making significant progress towards the clinic with our in vivo medicines in preclinical development for people living with serious diseases,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We look forward to sharing further proof of concept data at ASGCT, including preclinical data confirming our ability to increase the level of a protein to address diseases caused by loss of function or deleterious mutations via our differentiated gene upregulation editing strategy. Our progress with tLNP delivery highlights the potential to execute our gene upregulation strategy across multiple tissues with our ‘plug ‘n play’ approach.”

The complete list of Editas Medicine presentations is below. Abstracts can be accessed on the ASGCT website, and the presentations will be posted on the Editas Medicine website during the conference.

Oral Presentation:
Title: In Vivo Delivery of HBG1/2 Promoter Editing Cargo to HSC of Humanized Mouse and Non-Human Primate with Lipid Nanoparticles
Session Date and Time: Wednesday, May 14, 2025, 1:30 p.m. – 1:45 p.m. CT
Session Title: Translational Applications of Base and Prime Editors
Room: 265-268
Final Abstract Number: AMA353

Poster Presentations:
Title: Design and Development of Improved LNP Targeting Ligands for In Vivo Hematopoietic Stem Cell Editing
Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
Session Title: Tuesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA245

Title: Design of Chemically Modified AsCas12a Guide RNAs for Increased Potency of LNP-Delivered Gene Editing Cargos
Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
Session Title: Tuesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA420

Title: In Vivo Gene Editing and Disease-Associated Biomarker Reduction for Multiple Liver Targets in Non-human Primate Using AsCas12a Nuclease Delivered by LNP
Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
Session Title: Wednesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA640

Title: In Vivo CRISPR Editing of Genetic Regulatory Regions Results in Functional Upregulation of Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice
Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
Session Title: Wednesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA351

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 in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, 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.

Media and Investor Contact:
ir@editasmed.com  

FAQ

What new gene editing data will Editas Medicine (EDIT) present at ASGCT 2025?

Editas Medicine will present 5 abstracts at ASGCT 2025, including one oral presentation on in vivo preclinical data using targeted lipid nanoparticles for HBG1/2 promoter editing, and four poster presentations showing preclinical results for liver targeting and gene upregulation strategies.

When and where is Editas Medicine (EDIT) presenting at the ASGCT 2025 conference?

Editas Medicine will present at the 28th Annual ASGCT Meeting from May 13-17, 2025, in New Orleans, LA. The oral presentation is scheduled for May 14, 2025, at 1:30 PM CT, with poster sessions on May 13 and May 14, 2025.

What are the key findings from Editas Medicine's (EDIT) liver targeting research?

Editas Medicine demonstrated successful in vivo gene editing in the liver with high target editing levels and biomarker response, using AsCas12a mRNA and modified guide RNAs delivered through Genevant's LNP technology.

How is Editas Medicine (EDIT) advancing its gene upregulation strategy?

Editas Medicine showed proof of concept data for upregulating target protein expression in the liver, reducing disease-associated biomarkers in mouse models, and developing a 'plug 'n play' approach that can be used across multiple tissues.

What new delivery methods is Editas Medicine (EDIT) developing for gene editing?

Editas Medicine is developing targeted lipid nanoparticles (tLNPs) for delivering editing cargo to blood stem cells, and using Genevant's LNP technology for liver targeting, with improvements in targeting ligands and guide RNA modifications for better editing outcomes.