SLEEK enables high efficiency, multi-transgene knock-in of induced Pluripotent Stem Cells (iPSCs), T cells, and Natural Killer (NK) cells
Data support SLEEK as an optimized approach to develop next generation cell therapy medicines
CAMBRIDGE, Mass., Aug. 20, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced data on a new gene editing technology termed SLEEK (SeLection by Essential-gene Exon Knock-in). The Company reported these data in an oral presentation at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, being held virtually August 18-20, 2021.
Despite major progress in achieving gene disruption, efficient knock-in of transgenes continues to be a significant challenge for the gene editing field. To solve this challenge, SLEEK was developed enabling high knock-in efficiencies with different transgenes while also ensuring robust, transgene expression. Editas Medicine believes that SLEEK may enable the development of next generation cell therapeutics for cancer and other serious diseases.
New preclinical data demonstrated that SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that selects for cells containing the knock-in cargo. In addition, high percentage knock-in efficiencies were enabled by Editas Medicine’s proprietary engineered AsCas12a nuclease. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including iPSCs, T cells, and NK cells. Additionally, SLEEK may be used to fine-tune the expression levels of transgene cargos, an important attribute of next-generation cell therapy medicines.
“We find the new gene editing SLEEK technology to have immense potential, as it enables nearly 100 percent knock-in of functional transgene cargos at specific locations in the genome, which we believe to be the highest in the gene editing field across multiple cell types. We believe that this novel technology has broad applications and may result in substantially improved gene edited cell medicines, including for novel CAR-T and CAR-NK cell therapies,” said Mark S. Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We believe SLEEK is an optimal approach to achieve highly efficient multi-transgene knock-in for the next generation of cell therapy medicines, and we are leveraging this technology across many oncology programs, including for treatment of a variety of solid tumors.”
In addition to the SLEEK oral presentation, the Company also presented a poster on its CALITAS algorithm and software. CALITAS is a new, state-of-the-art, CRISPR-Cas tuned, DNA aligner, and is useful for the identification of potential off-target sites in silico. Editas Medicine made CALITAS freely available to the scientific community at github.com/editasmedicine/calitas.
Full details of the Editas Medicine presentations at the Cold Spring Harbor Laboratory meeting can be accessed in the Posters & Presentations section on the Company’s website.
About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) 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. For the latest information and scientific presentations, please visit www.editasmedicine.com.
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 pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical 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 represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.
Read more here: Source link