Full Press Release Details
Cornell Medical College and Cellectis Announce Research Alliance
Advancing Drug Discovery and the Translation of Novel Immunotherapies in
Will Focus on Improving Patient Outcomes in AML Using Targeted Cellular
Therapy Developed by Cellectis
NEW YORK--(BUSINESS WIRE)--June 2, 2015--Regulatory News:
Weill Cornell Medical College and Cellectis (Paris:ALCLS) have entered
into a strategic translational research alliance to accelerate the
development of a targeted immunotherapy for patients with acute
myelogenous leukemia (AML), a deadly blood cancer. The alliance will
foster the development of Cellectis' lead product candidate in AML,
The collaboration combines Weill Cornell's broad expertise and resources
in translational stem cell science and developmental therapeutics with
Cellectis' work in development and manufacturing of gene edited CAR-T
cell product candidates, a special kind of immune cell that includes an
antibody-derived receptor. The research will be led by co-principal
investigators Dr. Gail J. Roboz, director of the leukemia program and an
associate professor of medicine at Weill Cornell, and Dr. Monica Guzman,
an assistant professor of pharmacology in medicine at Weill Cornell. Dr.
Roboz is an internationally recognized leader in the field of acute
leukemia and will design and implement clinical testing of UCART123 in
patients with AML. Dr. Guzman is a renowned leukemia stem cell biologist
who specializes in preclinical and early-stage testing to optimize the
development of stem cell-targeted cancer drugs.
The alliance will seek to accelerate the development of Cellectis'
UCART123 in AML. Cellectis' proprietary allogeneic CAR T-cell platform
utilizes T-cells (immune cells) from healthy donors. The T-cells are
engineered with a Chimeric Antigen Receptor (CAR), which enables them to
detect specific proteins (antigens) expressed on malignant tumors. Large
numbers of allogeneic CAR-modified T-cells are grown in the laboratory
and then infused into a patient. The enhanced cells are designed to
recognize and attack stem cells harboring the CD123 antigen, which is
present on AML blast and stem cells. To enhance safety and minimize
toxicity for patients, the company's gene-editing process features
customized control properties that seek to prevent the T cells from
inappropriately attacking healthy tissues. Cellectis hopes to develop a
cost-effective, "off-the-shelf" allogeneic CAR T-cell product, designed
for efficient storage and distribution to patients around the globe.
Cellectis in April opened a new research and development facility in New
York City, located in close proximity to the Weill Cornell campus.
"We are pleased to collaborate with Cellectis to develop and advance
next-generation treatments for patients with this devastating form of
leukemia," said Dr. Laurie H. Glimcher, the Stephen and Suzanne Weiss
Dean of Weill Cornell Medical College. "Cellectis' proficiency in genome
engineering and our complementary expertise in translational research
will help us realize our common goal of improving human health in New
York and around the globe."
"CAR-T cells have shown remarkable promise in the treatment of acute
lymphoblastic leukemia," Dr. Roboz said. "Cellectis has interesting
preclinical data on UCART123 and our alliance will seek to build on
these findings to better understand the clinical potential of this
therapy. Our patients are anxiously awaiting the start of clinical
"Weill Cornell offers unsurpassed expertise in translational research,
with a wealth of leading-edge technologies and resources to help advance
our pipeline of unique CAR-T product candidates," said Dr. Mathieu
Simon, executive vice president and chief operating officer at
Cellectis. "We are excited by the prospect of working with Dr. Roboz,
Dr. Guzman and other premier investigators in leukemia stem cell
Weill Cornell's Office of BioPharma Alliances and Research
Collaborations negotiated the three-year alliance. In the program's
pre-clinical phase, Weill Cornell researchers will perform multiple
analyses, including data mining of primary AML samples, immune profiling
of AML patients and in vitro evaluation of allogeneically derived
anti-CD123 CAR-T cells. In the alliance's second phase, Weill Cornell
and Cellectis will jointly develop protocols to facilitate early-phase
testing, including phase 1 clinical trials.
"Cellectis believes the CAR-T platform has the potential to transform
the way cancer patients are treated. We are confident that our broad,
cross-discipline collaboration with Weill Cornell will foster creativity
and speed in drug development for the benefit of clinicians and patients
living with AML," said Dr. Andr Choulika, chief executive officer of
The mission of Weill Cornell's Office of BioPharma Alliances and
Research Collaborations is to proactively generate, structure and market
translational research alliances with industry in order to advance
promising research projects that have commercial potential. For more
information, contact Larry Schlossman at las2041@med.cornell.edu
About Weill Cornell Medical College
Weill Cornell Medical College, Cornell University's medical school
located in New York City, is committed to excellence in research,
teaching, patient care and the advancement of the art and science of
medicine, locally, nationally and globally. Physicians and scientists of
Weill Cornell Medical College are engaged in cutting-edge research from
bench to bedside aimed at unlocking mysteries of the human body in
health and sickness and toward developing new treatments and prevention
strategies. In its commitment to global health and education, Weill
Cornell has a strong presence in places such as Qatar, Tanzania, Haiti,
Brazil, Austria and Turkey. Through the historic Weill Cornell Medical
College in Qatar, the Medical College is the first in the U.S. to offer
its M.D. degree overseas. Weill Cornell is the birthplace of many
medical advances-including the development of the Pap test for cervical
cancer, the synthesis of penicillin, the first successful embryo-biopsy
pregnancy and birth in the U.S., the first clinical trial of gene
therapy for Parkinson's disease, and most recently, the world's first
successful use of deep brain stimulation to treat a minimally conscious
brain-injured patient. Weill Cornell Medical College is affiliated with
NewYork-Presbyterian Hospital, where its faculty provides comprehensive
patient care at NewYork-Presbyterian Hospital/Weill Cornell Medical
Center. The Medical College is also affiliated with Houston Methodist.
For more information, visit weill.cornell.edu.
Cellectis is a preclinical stage biopharmaceutical company focused on
developing immunotherapies based on gene edited engineered CAR-T cells