Date: 2015-06-02
Type of information: R&D agreement
Compound: UCART123
Company: Cellectis (France) Weill Cornell Medical College (USA - NY)
Therapeutic area: Cancer - Oncology
Type agreement: R&D
Action mechanism: immunotherapy product/cell therapy/gene therapy. 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. 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.
Disease: acute myelogenous leukemia (AML)
Details:
- • On June 2, 2015, Weill Cornell Medical College and Cellectis have entered into a strategic translational research alliance to accelerate the development of a targeted immunotherapy for patients with acute myelogenous leukemia (AML). The alliance will foster the development of Cellectis’ lead product candidate in AML, UCART123.
- 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. 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 in April opened a new research and development facility in New York City, located in close proximity to the Weill Cornell campus.
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.
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