Editas Medicine Announces Publication in Nature Biotechnology of Comprehensive SLEEK Gene Editing Technology Data
May 01 2023 - 4:01PM
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome
editing company, today announced that the journal Nature
Biotechnology published the comprehensive data from a study of the
proprietary SLEEK (SeLection by Essential-gene Exon Knock-in) gene
editing technology.
Despite major progress in achieving gene disruption with
CRISPR-Cas gene editing technologies, efficient knock-in of
transgenes continues to be a significant challenge for the gene
editing field. To solve this challenge, SLEEK was developed to
enable high knock-in efficiency with both viral and non-viral
transgene formats while also ensuring robust simultaneous
expression of up to four transgene cargos.
The study demonstrated that utilizing SLEEK results in the
knock-in of multiple clinically relevant transgenes through a
proprietary process that specifically selects only those cells
containing the knock-in cargo. This process was developed by
leveraging Editas Medicine’s proprietary engineered AsCas12a
nuclease, which can achieve very high editing efficiency while
maintaining high specificity. More than 90 percent knock-in
efficiencies were observed in various clinically relevant target
cells, including T cells, B cells, iPSCs, and NK cells.
Additionally, SLEEK can be used to fine-tune the expression levels
of transgene cargos, an important feature of next-generation cell
therapies. As a demonstration of SLEEK’s potential value in
clinical applications, the study authors used SLEEK to generate
iPSC-derived NK cells capable of high-levels of in vivo persistence
and robust tumor clearance in a solid tumor animal model.
“We are thrilled Nature Biotechnology published our paper
sharing the comprehensive data on our SLEEK gene editing technology
as we believe SLEEK has immense potential for gene editing drug
development. As shared in the publication, SLEEK technology enables
nearly 100 percent knock-in of functional transgene cargos at
specific locations in the genome which may result in highly
efficient multi-transgene knock-in for the next generation of cell
therapy medicines,” said John A. Zuris, Ph.D., Director of Editing
Technologies, Editas Medicine, and senior author on the study.
Editas Medicine believes that SLEEK may enable better product
purity as well as shorter manufacturing timelines for the next
generation of cell therapy medicines. Earlier this year, the
Company announced it licensed its interest in the SLEEK technology
to Shoreline Biosciences for specific usage in iPSC-derived NK and
iPSC-derived macrophage cell therapies for oncology. The SLEEK
technology remains an important Editas capability in iPSC
engineering for a wide variety of future applications.
About Editas MedicineAs a
clinical stage genome editing company, Editas Medicine is focused
on translating the power and potential of the CRISPR/Cas9 and
CRISPR/Cas12a 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 and Harvard University’s Cas9 patent
estates and Broad Institute’s Cas12a patent estate for human
medicines. For the latest information and scientific presentations,
please visit www.editasmedicine.com.
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Media and Investor Contact:
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
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