Editas Medicine Reports New In Vivo Proof of Concept Data in an Undisclosed Liver Target at the American Society of Gene and Cell Therapy Annual Meeting
In vivo CRISPR Editing Results in Functional Upregulation of a Liver Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice
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:
- 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.
- 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
Additional data on the undisclosed liver target will be shared in an oral presentation on
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:
Session Title: Wednesday Poster Reception
Final Abstract Number: AMA351
Additional
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:
Session Title: Translational Applications of Base and Prime Editors
Room: 265-268
Final Abstract Number: AMA353
Poster Presentations:
Title:
Session Date and Time:
Session Title: Tuesday Poster Reception
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:
Session Title: Tuesday Poster Reception
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:
Session Title: Wednesday Poster Reception
Final Abstract Number: AMA640
About Editas Medicine
As a pioneering gene editing company,
Media and Investor Contact: ir@editasmed.com
Source: Editas Medicine, Inc.
