Editas Medicine Presents Data on SLEEK Gene Editing Technology at the American Society of Gene and Cell Therapy Annual Meeting
SLEEK results in highly efficient multi-transgene knock-in and enables tunability of transgene expression
Double knock-in iNK cells demonstrate strong tumor killing effect and prolonged in vivo persistence in an ovarian cancer mouse model
“Despite recent progress, many cell-based medicines suffer from inefficient knock-in of transgene cargos and high heterogeneity that may reduce efficacy and pose potential safety issues. SLEEK is designed to address these challenges, by achieving efficient knock-in of multiple transgenes, avoiding silencing of transgene cargos over time, and enabling efficient knock-in using non-viral DNA donor templates,” said
SLEEK was used to insert CD16 and mbIL-15 cargos into iPSCs which generated NK cells with significantly improved tumor killing and survival compared to wild-type NK cells. SLEEK-edited cells also demonstrated strong antibody-dependent cellular cytotoxicity and improved persistence, without the need for exogenous cytokine support.
Further key findings presented about SLEEK include:
- Enables knock-in editing efficiencies of over 90% in relevant cell types, without impacting long-term viability or expansion.
- Permits constitutive and robust expression in iPSC-derived cell types where transgene promoter silencing has been a major challenge during the differentiation process.
- Demonstrated utility with clinically important cargos including chimeric antigen receptors (CARs) and allogeneic shields.
- Provides targeted knock-in to clinically important ex vivo cell types including T cells, B cells, and NK cells.
- Allows for tunability of transgene expression based on different promoter strengths of various essential genes.
- Enables use of non-viral DNA templates matching performance of the AAV6 gold standard on key attributes, potentially supporting more complex knock-in cassettes by avoiding AA6 cargo capacity limits.
Additional Presentations at ASGCT
In an oral presentation at the ASGCT Annual Meeting on
On
The complete list of
Oral Presentations:
Title: Exploratory Immuno-Safety Profile of EDIT-101, a First-in-Human In
Session Date and Time:
Presentation Time:
Session title: Gene and Cell Therapy Trials in Progress
Title: SLEEK: A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next-generation Cell-based Medicines
Session Date and Time:
Presentation Time:
Session title: New Gene Editing Technologies and Applications
Title: A Mutation-Independent CRISPR/Cas9-Based ʻKnockout and Replaceʼ Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
Session Date and Time:
Presentation Time:
Session title: Ophthalmic and Auditory Diseases
Poster Presentation:
Title: EDIT-202, A Multiplexed CRISPR-Cas12a Gene-Edited iPSC-Derived NK Cell Therapy has Prolonged Persistence, Promotes High Cytotoxicity, and Enhances In
Session Date and Time:
Session Title: Cancer - Targeted Gene and Cell Therapy II
About SLEEK Gene Editing
SLEEK (SeLection by Essential-gene Exon Knock-in) gene editing is an optimized approach to developing the next generation of cell therapy medicines for cancer and other serious diseases. Utilizing Editas Medicine’s proprietary engineered AsCas12a nuclease, SLEEK enables high efficiency, multi-transgene knock-in of induced pluripotent stem cells (iPSCs), T cells, and natural killer (NK) cells while ensuring robust, transgene expression.
About Editas Medicine
As a leading genome 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. 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 factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. 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.
Contacts: MediaCristi Barnett (617) 401-0113 cristi.barnett@editasmed.com InvestorsRon Moldaver (617) 401-9052 ir@editasmed.com
Source: Editas Medicine, Inc.
