Research includes first-ever application of AsCas12a in vivooptimized LNP delivery, and modification of gene-editing RNA guides.
Data supporting our development in vivo Gene editing drug pipeline
CAMBRIDGE, Mass., May 10, 2024 (Globe Newswire) — Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced preclinical data demonstrating several capabilities. announced. in vivo Capacity for innovative development in vivo Gene editing drugs. The company plans to present these data later today in an oral presentation and virtually at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore.
“Editas has made great strides in achieving its vision of becoming an industry leader. in vivo Programmable gene editing medicine. Our team has demonstrated multiple scientific advances, including lipid nanoparticle (LNP) formulations, to advance efficient formulations. in vivo AsCas12a messenger RNA (mRNA) delivery and gene editing by AsCas12 nuclease in vivo, a science-first technology that guides RNA modifications to increase the efficacy of gene editing,” said Linda C. Barkley, Ph.D., Chief Scientific Officer of Editas Medicine. “Our preclinical data underscore our therapeutic promise. in vivo This is an important step toward, and confirming, the capability for a robust pipeline of gene-edited drugs. in vivo We plan to have a proof of concept by the end of the year. ”
Data highlights:
- We developed and optimized LNP specifically for the following purposes: in vivo Gene editing using lipid formulations that potently transfect the ocular trabecular meshwork.
- Identify modifications to guide RNA that improve in vivo Editing efficiency.
- We developed a mouse model of myocilin-associated primary open-angle glaucoma (POAG) in which expression of human mutant myocilin increases intraocular pressure (IOP).
- Proven efficacy with AsCas12a nuclease in vivo.
- Administration of optimized LNPs delivering AsCas12a mRNA and optimized gRNA to the POAG model reduced IOP at all doses tested.
The company also presented two poster presentations demonstrating further scientific advances aimed at improvement. in vivo Gene editing.
A complete list of Editas Medicine ASGCT presentations is listed below, and posters and presentation materials are available on the Editas Medicine website.
Oral presentation:
title: LNP-based CRISPR/Cas12a delivery for potential treatment of myocilin-associated glaucoma
Session date and time: Friday, May 10, 2024, 3:45pm – 5:30pm ET
Presentation time: 4pm to 4:15pm
Session title: Advances in in vivo gene therapy technology
room: Room 324-326
Final abstract number: 276
Poster presentation:
title: Chemically modified AsCas12a guide RNA improves lipid nanoparticle mediation in vivo Gene editing in various tissues
Session date and time: Thursday, May 9, 2024, 12:00 PM ET
Session title: Thursday Poster: Gene Disruption and Excision
Presentation room: exhibition hall
Final abstract number: 1182
title: Metagenomic discovery and screening of novel recombinase proteins for targeted integration
Session date and time: Friday, May 10, 2024, 12:00 PM ET
Session title: Friday Poster: Targeted Gene Insertion
Presentation room: exhibition hall
Final abstract number: 1681
about editas medicine
As a clinical-stage gene editing company, Editas Medicine is leveraging the power and potential of CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust treatment pipeline for people living with serious diseases around the world. The focus is on embedding. Editas Medicine aims to discover, develop, manufacture and commercialize innovative, durable and precision genomic medicines for a wide range of diseases. Editas Medicine is the exclusive licensee of the Broad Institute's Cas12a patent assets and his Cas9 patent assets for human medicines from the Broad Institute and Harvard University. For the latest information and scientific publications, please visit www.editasmedicine.com.
Forward-looking statements
This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995. The words “anticipated,” “believed,” “continue,” “may,” and “estimate,” “expect,” “intend,” “may,” and “plan.” The words “may,” “anticipate,” “plan,” “goal,” “,” “should,” “will” and similar expressions are intended to identify forward-looking statements. However, not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Company's preclinical studies and its research and development programs, and the timing of the Company's receipt and presentation of data from the preclinical studies. It will be. in vivo You should not place undue reliance on these forward-looking statements, as we may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements. Actual results and events may differ from these forward-looking statements as a result of a variety of important factors, including the uncertainties inherent in the initiation and completion of preclinical studies and the availability and timing of results from preclinical studies. may differ materially from the plans, intentions and expectations disclosed herein. -Clinical research. These and other risks are described in more detail under the caption “Risk Factors” in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. the Securities and Exchange Commission and other filings that we may make with the Securities and Exchange Commission in the future. The forward-looking statements contained in this press release speak only as of the date of this press release and the Company expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise. I deny it.