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, will present first data exploring novel TALEN® editing
processes in hematopoietic stem and progenitor cells (HSPCs) at the
American Society of Gene and Cell Therapy (ASGCT) being held on May
7-11, 2024.
“These two posters showcase the potential and
versatility of the TALEN® technology to promote efficient gene
insertion in HSPCs. We show that circular single strand DNA
templates can be efficiently delivered to HSPCs and enable
unprecedented efficiency of gene insertion without compromising the
viability, fitness and differentiation capacity of edited cells”
commented Julien Valton, Ph.D., Vice President of Gene Therapy at
Cellectis.
“We also illustrate a novel TALEN® mediated-DNA
template insertion approach that rewires the natural ability of
myeloid cells to cross the blood brain barrier to efficiently
vectorize a genetically encoded-therapeutic protein to the brain.
This approach is, by essence, versatile and could be used to
vectorize an array of therapeutic proteins to the brain and
potentially address multiple neurological disorders.”
Poster presentation: Intron Editing of
HSPC Enables Lineage-Specific Expression of
Therapeutics
Gene therapy using edited hematopoietic and
progenitor stem cells (HSPCs) has the potential to provide a
lifelong supply of genetically encoded therapeutics.
Today, most therapies are impacted with the
difficulty to cross the blood-brain barrier (BBB). The BBB is a
continuous endothelial membrane that, along with pericytes and
other components of the neurovascular unit, limits the entry of
toxins, pathogens, protein and small molecules to the brain.
Cellectis has developed a TALEN® mediated
promoter-less intron editing technology that enables the expression
of a therapeutic transgene exclusively by monocyte derived from
edited HSPCs.
The edited cells containing genetically encoded
therapeutic proteins have the capacity to cross the blood-brain
barrier and secrete the corresponding therapeutic within the
brain.
This novel editing approach is an important
addition to the HSPC gene editing toolbox that might unlock new
strategies for the treatment of metabolic and neurological
diseases.
Research data showed that:
- Intron editing can be performed
within B-cell, T-cell, Monocyte-specific endogenous genes (CD20,
CD4 and CD11b, respectively)
- Intron editing allows expression of
transgenes in a lineage-specific manner without markedly impacting
the expression of the endogenous gene targeted
- Editing of CD11b intron using a
therapeutic transgene encoding IDUA (the enzyme missing in Type-1
Mucopolysaccharidosis patients) enables to restrict the expression
of IDUA to the myeloid lineage.
- Edited HSPCs efficiently engraft in
the bone-marrow of immunodeficient mice and differentiate into
edited myeloid cells that can cross the BBB and populate the
brain.
- The intron editing strategy
described in this work is versatile and could be potentially used
to vectorize multiple genetically encoded-therapeutic proteins to
the brain and thus address multiple metabolic and neurological
disorders.
Title: Intron Editing
of HSPC Enables Lineage-Specific Expression of
Therapeutics
Presenter: Julien Valton, Ph.D., Vice President
Gene Therapy at Cellectis Session Date/Time:
May 5, 2024 at 12PM ET Session Title: Gene
Targeting and Gene Correction New
Technologies Presentation Room: Exhibit
Hall Final Abstract Number:
721
Poster presentation: Circularization of
Non-Viral Single-Strand DNA Template for Gene Correction and Gene
Insertion Improves Editing Outcomes in HSPCs
Today, most of the gene insertion approaches
used to edit HSPCs ex vivo are hampered by the low efficiency of
DNA template delivery into their nucleus.
Cellectis has developed and optimized a novel
gene editing process, leveraging the TALEN® technology and circular
single strand DNA template delivery, enabling highly efficient gene
insertion in HSPCs.
Research data showed that:
- Non-viral single strand DNA
delivery associated to TALEN® technology allows gene insertion in
long-term repopulating hematopoietic stem cells
- Circularization of the single
strand DNA further increases the rates of gene insertion without
impacting cellular viability and fitness of HSPCs, facilitating the
development of next generation of ex vivo cell therapies
Title: Circularization of Non-Viral Single-Strand DNA
Template for Gene Correction and Gene Insertion Improves Editing
Outcomes in HSPCs
Presenter: Alex Boyne, Gene Editing Platform
Manager at Cellectis Session Date/Time: May
9, 2024 at 12PM ET Session Title: Nonviral
Therapeutic Gene Delivery and Synthetic/Molecular
Conjugates Presentation Room: Exhibit
Hall Final Abstract Number: 1235
Full abstracts and poster presentations will be available on
Cellectis’ website following the event:
https://www.cellectis.com/en/investors/scientific-presentations/
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 Statement
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
“might,” “could,” “has the potential,” and “potentially,” 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. Forward-looking statements include statements about
the potential of our R&D 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
contacts:
Pascalyne Wilson, Director, Communications, +33
(0)7 76 99 14 33, media@cellectis.com
Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7
76 77 46 93,
Investor Relation
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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