Cellectis Publishes a Scientific Article Unveiling Three Key Factors for Efficient TALE Base Editing
21 June 2024 - 6:30AM
Cellectis Publishes a Scientific Article Unveiling Three Key
Factors for Efficient TALE Base Editing
Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ:
CLLS), a clinical-stage biotechnology company using its pioneering
gene-editing platform to develop life-saving cell and gene
therapies, announced today the publication of a manuscript in
Scientific Reports, demonstrating how three key factors can be
determinant for efficient TALE base editing.
TALE base editors are a recent and important
addition to the gene editing landscape. By design, TALE base
editors do not create break within DNA strands as does CRISPR/Cas9,
or other engineered nucleases, and is a promising therapeutic
strategy for genetic diseases. A key aspect to broaden the scope of
possible applications is our comprehension of design rules.
TALE base editors rely on the deamination of
cytidines within double strand DNA, leading to the formation of an
uracil (U) intermediate. These molecular tools are fusions of
transcription activator-like effector domains (TALE) for specific
DNA sequence binding, split-DddA deaminase halves that will, upon
catalytic domain reconstitution, initiate the conversion of a
cytosine (C) to a thymine (T), and an uracil glycosylase inhibitor
(UGI).
Previous works have pointed towards the
positioning of targeted cytosine to be a key determinant for
efficient editing.
To extend the understanding of key determining
factors allowing efficient TALE base editing (C-to-T conversion),
Cellectis investigated whether the nature (length and composition)
of the linker that connects the TALE array with the split deaminase
catalytic heads could impact C-to-T conversion within the editing
window.
The datasets presented in this paper highlight
how three key factors, spacer length, TALEB architecture and
composition of the surrounding bases, can impact editing outcomes
and further improve our understanding of TALE base editors'
activity and specificity, leading to the possibility to tune and
control editing using educated designs.
“This experimental strategy used by Cellectis to
characterize editing profiles in depth and in a high throughput
format could easily be applied to any new editors to continue
expanding this platform for potential therapeutic applications,”
said Maria Feola, Scientist III, Manager, Gene Editing at
Cellectis.
Research data specifically underlines the
primordial importance of the positions preceding the targeted TC,
which markedly increases editing efficiency.
The article is available on Scientific Reports
website by clicking on this
link: https://www.nature.com/articles/s41598-024-63203-8
About
Cellectis
Cellectis is a clinical-stage biotechnology
company using its pioneering gene-editing platform to develop
life-saving cell and gene therapies. Cellectis utilizes an
allogeneic approach for CAR-T immunotherapies in oncology,
pioneering the concept of off-the-shelf and ready-to-use
gene-edited CAR T-cells to treat cancer patients, and a platform to
make therapeutic gene editing in hemopoietic stem cells for various
diseases. As a clinical-stage biopharmaceutical company with over
24 years of experience and expertise in gene editing, Cellectis is
developing life-changing product candidates utilizing TALEN®, its
gene editing technology, and PulseAgile, its pioneering
electroporation system to harness the power of the immune system in
order to treat diseases with unmet medical needs. Cellectis’
headquarters are in Paris, France, with locations in New York, New
York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq
Global Market (ticker: CLLS) and on Euronext Growth (ticker:
ALCLS).
Forward-looking
Statements
This press release contains “forward-looking”
statements within the meaning of applicable securities laws,
including the Private Securities Litigation Reform Act of 1995.
Forward-looking statements may be identified by words such as
“promising,” “could,” and “potential,” or the negative of these and
similar expressions. These forward-looking statements, which are
based on our management’s current expectations and assumptions and
on information currently available to management, include
statements about the potential benefit and potential development of
the Company’s research programs. These forward-looking statements
are made in light of information currently available to us and are
subject to numerous risks and uncertainties, including with respect
to the numerous risks associated with biopharmaceutical product
candidate development. Furthermore, many other important factors,
including those described in our Annual Report on Form 20-F and the
financial report (including the management report) for the year
ended December 31, 2023 and subsequent filings Cellectis makes with
the Securities Exchange Commission from time to time, as well as
other known and unknown risks and uncertainties may adversely
affect such forward-looking statements and cause our actual
results, performance or achievements to be materially different
from those expressed or implied by the forward-looking statements.
Except as required by law, we assume no obligation to update these
forward-looking statements publicly, or to update the reasons why
actual results could differ materially from those anticipated in
the forward-looking statements, even if new information becomes
available in the future.
For further information on Cellectis, please
contact:
Media contacts:Pascalyne Wilson, Director,
Communications, + 33 (0)7 76 99 14 33, media@cellectis.comPatricia
Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46
93
Investor Relations contacts:Arthur Stril,
Interim Chief Financial Officer, +1 (347) 809 5980,
investors@cellectis.comAshley R. Robinson, LifeSci Advisors, +1
617 430 7577
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