The Company will report these data later today in an oral presentation at Annual Meeting of the
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
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
About
As a clinical-stage gene editing company,
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 anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although 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 for the Company s receipt and presentation of data from its preclinical studies, including confirming in vivo proof-of-concept by the end of 2024. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including uncertainties inherent in the initiation and completion of pre-clinical studies and availability and timing of results from pre-clinical studies. These and other risks are described in greater detail under the caption Risk Factors included in the Company s most recent Annual Report on Form 10-K, which is on file with the
Contact:
Tel: (617) 401-0113
Email: cristi.barnett@editasmed.com
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