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Cellectis and Weill Cornell collaborate to research new leukemia immunotherapies

French biopharmaceutical firm Cellectis has entered into a strategic translational research alliance with Weill Cornell Medical College in the US to accelerate the development of a targeted immunotherapy for patients with acute myelogenous leukemia (AML).

The research collaboration will advance the development of the French firm’s lead product candidate in AML, called UCART123.

The deal will combine Weill Cornell’s 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.

Weill Cornell leukemia program director Dr Gail Roboz and assistant professor of pharmacology in medicine Dr Monica Guzman will be responsible for overseeing the research project.

Dr Roboz will design and implement clinical testing of UCART123 in patients with AML, while Dr Guzman specializes in preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

Cellectis executive vice-president and chief operating officer Dr Mathieu Simon said: "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."

The company’s allogeneic CAR T-cell platform uses T-cells (immune cells) from healthy donors. The cells are designed with a Chimeric Antigen Receptor (CAR), which allows them to detect specific proteins (antigens) expressed on malignant tumors.

Large numbers of allogeneic CAR-modified T-cells are grown in the laboratory and infused into a patient, to recognize and attack stem cells harboring the CD123 antigen, which is present on AML blast and stem cells.

Dr. Roboz said: "CAR-T cells have shown remarkable promise in the treatment of acute lymphoblastic leukemia.

"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 trials."

Cellectis opened a new research and development facility in New York City, in close proximity to the Weill Cornell campus, in April.