Editas Medicine Preclinical Data Highlights Advancement of in vivo Gene Editing Medicine Technologies at the American Society of Gene and Cell Therapy Annual Meeting
Editas Medicine showcased preclinical data at the American Society of Gene and Cell Therapy Annual Meeting, highlighting advancements in in vivo gene editing technologies. The research included the first-ever application of AsCas12a in vivo, optimized LNP delivery, and gene editing RNA guide modifications. The data supports the development of Editas Medicine's pipeline of in vivo gene editing medicines, demonstrating therapeutic promise and potential proof of concept by the end of the year.
Editas Medicine demonstrated significant scientific advances in in vivo gene editing technologies, including lipid nanoparticle formulations for efficient delivery and guide RNA modifications to enhance editing potency.
The efficacy of AsCas12a nuclease in vivo was demonstrated by Editas Medicine, showing a decrease in intraocular pressure in a model of myocilin-associated primary open-angle glaucoma.
The development of an optimized LNP specifically for in vivo gene editing and the identification of modifications to improve in vivo editing efficiency are promising advancements showcased in the preclinical data.
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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
Presentation Time: 4:00 - 4:15 p.m.
Session title: Advancements in Technologies for In Vivo Gene Therapies
Room: Room 324-326
Final Abstract Number: 276
Poster Presentations:
Title: Chemically Modified AsCas12a Guide RNAs Improve Lipid Nanoparticle-Mediated In Vivo Gene Editing in Different Tissues
Session Date and Time: Thursday, May 9, 2024, 12:00 p.m. ET
Session Title: Thursday Posters: Gene Disruption and Excision
Presentation Room: Exhibit 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 p.m. ET
Session Title: Friday Posters: Targeted Gene Insertion
Presentation Room: Exhibit Hall
Final Abstract Number: 1681
About Editas Medicine
As a clinical-stage gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, 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 ‘‘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 Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.
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