UCARTs

 Universal Chimeric Antigene Receptors

 

UCART (Universal Chimeric Antigene Receptor T-cells) are “off-the-shelf” allogeneic products, whose production can be industrialized and thereby standardized with consistent pharmaceutical release criteria, over time and from batch to batch.

Our lead immuno-oncology product candidates, which we refer to as UCARTs, are all allogeneic CAR T-cells engineered to be used for treating any patient with a particular cancer type. Each UCART product candidate targets a selected tumor antigen and bears specific engineered attributes, such as compatibility with specific medical regimens that cancer patients may undergo. UCART is our first therapeutic product line that we are developing with our gene editing platform to address unmet medical needs in oncology.

UCART19

During the 57th American Society of Hematology (ASH) Annual Meeting on December 5, 2015, Great Ormond Street Hospital (GOSH) at University College London (UCL) presented encouraging data from a first-in-man use of UCART19 product candidate. This first-in-human application of our TALEN® engineered T-cell product candidate represents a landmark in the use of new gene engineering technology and provides early encouraging data for a ready-made T-cell strategy that will be further tested in clinical investigations. GOSH has treated in June 2015 a young leukemia patient under a special license from the Medicines & Healthcare products Regulatory Agency (MHRA) with Cellectis’ TALEN® gene edited allogeneic UCART19 product candidate because no other therapies were available for refractory relapsed acute lymphoblastic leukemia (ALL) following mismatched allogeneic stem cell transplantation. In response to an unsolicited request from Professor Waseem Qasim, Consultant Immunologist at GOSH and Professor of Cell and Gene Therapy at University College London (UCL) Institute of Child Health, Cellectis gave its approval for the use of its UCART19 product candidate and technologies under GOSH’s “Specials” license and responsibility, for the particular clinical needs of that individual patient.

On November 18, 2015, we signed with Servier an amendment to our collaboration agreement whereby notably Servier exercised its option to acquire the exclusive worldwide rights to further develop and commercialize UCART19, which was about to enter into Phase I clinical development for chronic lymphocytic leukemia (CLL) and acute lymphoblastic leukemia (ALL).

UCART123

Our first wholly-owned product candidate, UCART123, is an engineered T-cell product candidate that targets CD123, an antigen located on CD123 expressing leukemia such as on cancer cells in acute myeloid leukemia, or AML, and blastic plasmacytoid dendritic cell neoplasm, or BPDCN. UCART123 is at a preclinical stage of development. We have initiated the manufacturing process transfer of UCART123 to CELLforCURE, to whom we subcontract the manufacturing of the clinical supplies of UCART123, and we will start manufacturing clinical grade UCART123 in large scale according to GMP in 2016, for purposes of conducting clinical investigations. Preclinical and translational activities on UCART123 in AML will be performed in collaboration with Weill Cornell Medical College. The research at Weill Cornell is led by co-principal investigators Dr. Gail J. Roboz, Director of the leukemia program and an Associate Professor of medicine, and Dr. Monica Guzman, an Assistant Professor of pharmacology in medicine. In addition, we collaborate with the MD Anderson Cancer Center on the preclinical development of UCART123 in BPDCN in view of a potential clinical trial. The studies on UCART123 led at MD Anderson are under the direction of Pr. Hagop Kantarjian, MD, Chair, Department of leukemia.

UCART38 and UCARTCS1

UCARTCS1 and UCART38 are allogeneic engineered T-cell product candidates designed for the treatment of CS1-expressing or CD38-expressing hematologic malignancies which develop in multiple myeloma (MM). UCARTCS1 is at a preclinical stage of development. We intend to initiate manufacturing of UCARTCS1 according to GMP in 2016, for purposes of conducting clinical trials. Preclinical and translational activities for UCARTCS1 in MM will be performed in collaboration with the MD Anderson Cancer Center. UCART38 is at an early preclinical stage. Preclinical and translational activities on UCART38 in T-cell ALL are to be performed in collaboration with the MD Anderson Cancer Center.

UCART22

Like CD19, CD22 is a cell surface antigen expressed from the pre B-cell stage of development through mature B-cells. UCART22 is an allogeneic engineered T-cell product candidate designed for the treatment of acute lymphoblastic leukemia. UCART22 is at an early preclinical stage of development. Preclinical and translational activities on UCART22 in ALL will be performed in collaboration with the MD Anderson Cancer Center in view of a potential clinical trial.