Cellectis Presents Initial Preclinical Data on Two Novel Gene
Therapies for Patients with RAG1 Severe Combined Immunodeficiency
(SCID) and Hyper IgE syndrome at ESGCT 2021
Cellectis S.A. (NASDAQ: CLLS – EURONEXT GROWTH: ALCLS) (the
“Company”), a gene-editing company with clinical-stage
immuno-oncology programs using allogeneic chimeric antigen receptor
(CAR)-T cells and gene therapy programs for genetic diseases, in
collaboration with Professor Toni Cathomen, scientific director at
the Center for Chronic Immunodeficiency Medical Center at the
University of Freiburg, Germany, will present two oral
presentations at the European Society of Gene and Cell Therapy
(ESGCT) Congress to be held virtually from October 19-22, 2021.
Professor Cathomen’s team at
University of Freiburg will
be presenting encouraging pre-clinical data that supports
further evaluation of Cellectis’ .HEAL
platform, an innovative gene
therapy platform that uses a genome editing
approach based
on TALEN® , for two product
candidates targeting primary immunodeficiencies:
RAG1 for Severe Combined Immunodeficiency (SCID)
and STAT3
for Hyper IgE syndrome.
‘The data accepted for presentation at ESGCT
reflects our ongoing commitment to finding new ways to treat and
potentially provide a cure to patients that have failed to respond
to standard therapies. Utilizing Cellectis’ TALEN® technology,
which we believe to be the most precise, versatile, and effective
gene editing tool currently available, we demonstrate our potential
to precisely correct RAG1 and STAT3 deficient genes and restore
functionalities of the gene. These new milestones bring us one step
closer to our goal of unlocking the full potential of our gene
editing platform and bringing new therapies to patients with unmet
medical needs.’ said Philippe Duchateau, Ph.D, Chief Scientific
Officer of Cellectis.
Last
May, during Cellectis’ Innovation Days, the
Company revealed its new .HEAL platform, a novel hematopoietic
stem cell gene therapy that aims to address debilitating genetic
diseases. .HEAL leverages the power of TALEN® gene editing
technology to perform genome surgery, resulting in highly efficient
and precise gene inactivation, insertion, and correction in
hematopoietic stem cells (HSCs). Cellectis has announced ongoing
programs targeting sickle cell disease, lysosomal storage disorders
and primary immunodeficiencies.
Presentations details
Data presentation on preclinical
development of a TALEN® based
genome editing therapy for RAG1 deficiency
- Newborns with RAG1 SCID have
extremely low levels of B and T cells and a severe risk of
recurrent, life-threatening infections. RAG1 is an essential enzyme
specifically and temporarily expressed in the early development of
T and B cells, making traditional gene therapy approaches to treat
the disease challenging due to the need for tight and
precise spatio-temporal expression control.
- Previous attempts to treat the RAG1
deficiency via conventional gene therapy have produced
unsatisfactory results.
- These results highlight the need
for tight spatio-temporal control of RAG1 expression as key for
functional restoration and the use of a gene editing tool.
- Using Cellectis’ TALEN® technology
and .HEAL, Professor Cathomen engineered HSCs with a corrected copy
of RAG1 that replaced the existing, mutated copy of RAG1. The
precise replacement of the mutated gene enabled the corrected RAG1
gene to be expressed at its natural timing and stage of cell
development.
- 30% of gene correction was achieved
within the long-term HSC population.
The presentation titled ‘Preclinical development
of a TALEN based genome editing therapy for RAG1 deficiency’ will
be made on October 21 (9-11AM CET) by Manuel Rhiel, Ph.D University
of Freiburg, and can be found on the ESGCT website.
Presentation Details:
Data presentation on a preclinical
development of a TALEN® based
genome editing in T-cells for the treatment of Hyper-IgE-
Syndrome
- Hyper IgE syndrome is
a rare primary immunodeficiency disease that
clinically manifests as skin inflammation and recurrent skin and
lung infections. Mutations in the transcription factor STAT3 have
been associated with Hyper IgE. Alternative splicing gives rise to
two STAT3 isoforms, STAT3α and STAT3β that display distinct
functions.
- The α/β ratio needs to be tightly
regulated, which represents a major challenge for traditional gene
therapy approaches.
- Cellectis has developed a strategy
applicable in HSCs and T-cells to insert a corrected version of the
STAT3 gene into the patient’s genome to restore its
functionality.
- In T-cells isolated from patients,
60% integration was achieved. More importantly, the α/β isoforms
ratio was restored.
The presentation titled ‘Preclinical development
of a TALEN based genome editing in T cells for the treatment of
Hyper-IgE-Syndrome' will be made on October 20 (9-11AM CET) by
Viviane Dettmer, Ph.D, University of Freiburg, and can be found on
the ESGCT website.
About Cellectis Cellectis
is a gene editing company, developing first of its kind therapeutic
products. 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 21 years of 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. As part of its commitment to a cure,
Cellectis remains dedicated to its goal of providing lifesaving
UCART product candidates for multiple cancers including acute
myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL)
and multiple myeloma (MM). .HEAL is a new platform
focusing on hemopoietic stem cells to treat blood disorders,
immunodeficiencies and lysosomial storage
diseases. 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).
For more information,
visit www.cellectis.com Follow
Cellectis on social media: @cellectis, LinkedIn and
YouTube.
For further information, please
contact:
Media contacts: Margaret Gandolfo, Senior
Manager, Communications, +1 (646) 628 0300 Pascalyne
Wilson, Director, Communications, +33776991433,
media@cellectis.com
Investor Relation
contact: Eric Dutang, Chief Financial
Officer, +1 (646) 630
1748, investor@cellectis.com
Forward-looking Statements
This presentation 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 “at
this time,” “anticipate,” “believe,” “expect,” “on track,” “plan,”
“scheduled,” 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 our research and development projects and priorities, our
pre-clinical project development efforts and the timing of our
presentation of data. 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 as well as the duration and severity of the COVID-19
pandemic and governmental and regulatory measures implemented in
response to the evolving situation. With respect to our cash
runway, our operating plans, including product development plans,
may change as a result of various factors, including factors
currently unknown to us. 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, 2020 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.
- 20211019_ESGCT_press_release_ENGLISH_FINAL
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