In This Article:
In vivo CRISPR Editing Results in Functional Upregulation of a Liver Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice
CAMBRIDGE, Mass., May 13, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared in vivo proof of concept data supporting the development of a potentially first-in-class treatment for an undisclosed liver target in a poster presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans. Editas scientists will present the data in a poster session on Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT (6:30 p.m. – 8:00 p.m. ET).
An in vivo editing strategy using lipid nanoparticles (LNPs) with CRISPR/Cas RNA cargo was employed for an undisclosed liver target gene. The strategy mimics a naturally occurring, protective variant, resulting in upregulation of the target gene. This resulted in meaningful reduction in the clinically relevant disease-specific biomarker in mice.
Key findings include:
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An in vivo dose-response study in a disease-specific mouse model utilizing LNPs to deliver CRISPR/Cas-based cargo demonstrated maximal liver editing of the target gene (~70%) and resulted in robust target protein upregulation with >80% disease biomarker reduction.
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Editing and subsequent upregulated expression of the target gene in cynomolgus monkey hepatocytes treated with CRISPR/Cas-based editing cargo also achieved >50% target gene editing and >15-fold protein upregulation.
“This in vivo proof of concept data in an undisclosed liver disease target confirms our ability to achieve maximal target gene editing within hepatocytes and clinically meaningful reduction in disease biomarkers. We believe this therapeutic approach will be transformative in the future treatment of this disease,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We are making significant progress towards the clinic and look forward to sharing the disease target and our development candidate later this year.”
Additional data on the undisclosed liver target will be shared in an oral presentation on May 21 at TIDES USA 2025: Oligonucleotide & Peptide Therapeutics Conference in San Diego.
Poster Presentation Details:
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