Editas Medicine Preclinical Data Highlights Advancement of in vivo Gene Editing Medicine Technologies at the American Society of Gene and Cell Therapy Annual Meeting

Research includes the first-ever application of AsCas12a in vivo, optimized LNP delivery, and gene editing RNA guide modifications

Data to support development of the Company’s in vivo gene editing medicines pipeline  

CAMBRIDGE, Mass., May 10, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced the presentation of preclinical data demonstrating several in vivo capabilities towards developing transformative in vivo gene editing medicines. The Company will report these data later today in an oral presentation at Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Baltimore and virtually.

“Editas has made significant strides to achieve our vision of becoming a leader in in vivo programmable gene editing medicine. Our team has demonstrated multiple scientific advances, including lipid nanoparticle, or LNP, formulations to drive efficient in vivo delivery of AsCas12a messenger RNA (mRNA) and gene editing by AsCas12 nuclease in vivo, a scientific first, and guide RNA modifications to increase gene editing potency,” said Linda C. Burkly, Ph.D., Chief Scientific Officer, Editas Medicine. “Our preclinical data underscore the therapeutic promise of our in vivo capabilities towards a robust pipeline of gene editing medicines and are an important step towards confirming in vivo proof of concept by the end of the year.”

Data highlights:

• Developed and optimized an LNP specifically for in vivo gene editing with a lipid formulation that robustly transfects the ocular trabecular meshwork.
• Identified modifications to the guide RNA that improve in vivo editing efficiency.
• Developed a mouse model of myocilin-associated primary open angle glaucoma (POAG) where expression of human mutant myocilin results in elevated intraocular pressure (IOP).
• Demonstrated efficacy with AsCas12a nuclease in vivo.
• Administered optimized LNP delivering AsCas12a mRNA and optimized gRNA into a POAG model resulting in a decrease in IOP at all doses tested.
  

The Company also presented two poster presentations, demonstrating additional scientific advances designed to improve in vivo gene editing.

The complete list of Editas Medicine ASGCT presentations are listed below, and posters and presentation materials are available on the Editas Medicine website.

Oral Presentation:
Title: LNP-Based Delivery of CRISPR/Cas12a for the Potential Treatment of Myocilin-Associated Glaucoma
Session Date and Time: Friday, May 10, 2024, 3:45 – 5:30 p.m. ET