Cellectis to Present Pre-Clinical Data on HSPC Gene Therapy Program and Comprehensive Analysis of TALE-BE at the ESGCT 30th Annual Congress
24 Octobre 2023 - 10:30PM
Cellectis to Present Pre-Clinical Data on HSPC Gene Therapy Program
and Comprehensive Analysis of TALE-BE at the ESGCT 30th Annual
Congress
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 that they will be showcasing
pre-clinical data on its program of gene therapy for HSPC as well
as comprehensive analysis of TALE-BE editing determinants at
the European Society of Gene and Cell Therapy (ESGCT) 30th annual
congress that will take place on October 24-27, 2023 in Brussels,
Belgium.
The data will be presented in three posters:
Intronic editing enables lineage specific
expression of therapeutics relevant for HSPC gene therapy (Poster
N°646)
Presenter: Eduardo Seclen,
Senior Scientist & Team Leader, Gene Editing
Date/Time: Wednesday October
25th from 18:15 to 19:30 and Thursday October 26th from 19:30 to
20:30
- Intronic editing enables lineage
specific expression of therapeutics relevant for HSPC gene
therapy.
- TALEN®-mediated intron editing of
the CD11b locus results in the lineage-specific expression of a
reporter transgene in myeloid cells, with negligible expression in
HSPC or other cellular subsets in vitro and in vivo.
- We believe this intron editing
approach could be disruptive in HSPC gene therapy and brain
delivery of multiple therapeutics.
TALEN editing coupled to non-viral DNA delivery
enables efficient correction of sickle cell mutation with minimal
transcriptional changes and low level of HBB KO (Poster N°380)
Presenter: Julien Valton, VP,
Gene Therapy
Date/Time: Wednesday October
25th from 18:15 to 19:30 and Thursday October 26th from 19:30 to
20:30
- Using a combination of scRNA
sequencing and multiple genomic read out methodologies, we
demonstrate that the mutant HBB gene can be efficiently corrected
in HSPCs by TALEN®-mediated gene editing coupled to non-viral gene
delivery (ssODN) with a low risk of generating β-thalassemic
RBCs.
- TALEN®-mediated HBB editing coupled
to non-viral gene delivery (ssODN) in SCD patients’ HSPCs led to a
lower activation of p53 response compared to viral gene delivery
(AAV), preserves the transcriptomic profile of edited HSPCs in
vitro and their engraftment capacity in vivo.
Comprehensive analysis of TALE-BE editing
determinant (Poster N°667)
Presenter: Maria Feola,
Scientist III, Manager, Gene Editing
Date/Time: Wednesday October
25th from 17:00 to 18:15 and Thursday October 26th from 20:30 to
21:30
- The robustness and versatility of
genome engineering strategies we developed allowed us to gain
in-depth insight of TALE-BE editing rules in cellulo and further
highlighted that the composition surrounding the TC to be edited
could strongly impact editing efficiencies. Therefore, educated
choice of the TALE-BE architecture and positioning on DNA could
either prevent target sequence limitations (increasing targetable
sequence space) or decrease, if not eliminate, bystander editing
within the editing window, allowing for more precise genome editing
outcomes.
- We believe that the knowledge
presented will help ensure that genome editing-based strategies are
skillfully designed to minimize the risk of potential genotoxic
events, overall expanding the potential of TALE-BE for nuclear and
mitochondrial therapeutic cell engineering.
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
23 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
“believe”, “could”, “can” and “will,” 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 of the Company’s research and
pre-clinical 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, 2022 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
contact: Patricia Sosa Navarro, Chief of Staff
to the CEO, +33 (0)7 76 77 46 93, media@cellectis.com
Investor Relations
contacts: Arthur Stril, Chief Business Officer,
+1 (347) 809 5980, investors@cellectis.com Ashley R.
Robinson, LifeSci Advisors, +1 617 430 7577
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