Editas Medicine Showcases Gene Editing Advances at ASGCT Annual Meeting

Editas Medicine, Inc. (Nasdaq: EDIT) recently unveiled preclinical data showcasing significant advancements in in vivo gene editing. These findings were disclosed during an oral presentation at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Baltimore.

Editas Medicine, a frontrunner in the clinical-stage gene editing sector, has made considerable progress in developing in vivo gene editing medicines. The company's latest research includes the first-ever in vivo application of the AsCas12a gene editing system, optimized lipid nanoparticle (LNP) delivery mechanisms, and modified gene editing RNA guides. These innovations underline the potential of Editas Medicine's in vivo gene editing capabilities, laying the groundwork for a promising pipeline of gene editing therapeutics.

Linda C. Burkly, Ph.D., Chief Scientific Officer at Editas Medicine, highlighted the significance of these scientific milestones. She emphasized the company's progress in optimizing LNP formulations to facilitate efficient in vivo delivery of AsCas12a messenger RNA (mRNA) and gene editing using the AsCas12 nuclease. These developments represent a significant leap toward the company's goal of becoming a leader in in vivo programmable gene editing medicine.

Key data points from the research include the development and optimization of an LNP tailored for in vivo gene editing, utilizing a lipid formulation that effectively transfects the ocular trabecular meshwork. Additionally, the company identified specific modifications to the guide RNA that enhance in vivo editing efficiency. A mouse model of myocilin-associated primary open-angle glaucoma (POAG) was also developed, where the expression of human mutant myocilin leads to elevated intraocular pressure (IOP). The AsCas12a nuclease demonstrated efficacy in vivo, and the administration of the optimized LNP delivering AsCas12a mRNA and modified gRNA in the POAG model resulted in a reduction of IOP across all tested doses.

Further scientific accomplishments were showcased in two poster presentations, elaborating on additional advancements designed to improve in vivo gene editing.

Editas Medicine is dedicated to harnessing the power of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems. The company's mission is to translate these technologies into a comprehensive pipeline of treatments for serious diseases globally. As the exclusive licensee of the Cas12a patent estate from the Broad Institute and the Cas9 patent estates from the Broad Institute and Harvard University for human medicines, Editas Medicine is positioned at the forefront of genomic medicine innovation.