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August 18, 2017

Cellectis Starts BPDCN Phase I Trial with Gene-Edited CAR-T Therapy

  • Cellectis reported that the first patient has been dosed in a Phase I clinical trial evaluating its TALEN® gene-edited chimeric antigen receptor (CAR) T-cell product UCART123 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The BPDCN study follows on from the start in June of a Phase I study to evaluate the safety and efficacy of UCART123 in patients with acute myeloid leukemia (AML). Cellectis  says UCART123 is the first allogeneic, off-the-shelf, gene-edited CAR T-cell program targeting CD123 to enter clinical trials in the U.S.

    BPDCN is a rare, aggressive hematological cancer classified as an acute leukemia, for which there is no standardized therapeutic approach. Most BPDCN patients may achieve transient responses when treated using combination chemotherapy regimens for acute leukemia or lymphoma, but then they relapse. The new Phase I UCART123 study, carried out the MD Anderson Cancer Center, will evaluate the cell therapy in BPDCN patients in the relapsed, refractory, and front-line settings. 

    “We are eager to progress through clinical trials with UCART123, Cellectis’ wholly controlled gene-edited product candidate, next with the treatment of BPDCN, a rare but aggressive entity,” said  Loan Hoang-Sayag, M.D., Cellectis’ CMO. “With this innovative treatment, the hope is that our ‘off-the-shelf' approach will transform the way we think about cancer care and serve as the next step in curing this disease through the power of gene editing.”

    Cellectis’ lead allogeneic UCART (universal antigen receptor T-cells) product UCART19 is a TALEN (transcription activator-like effector nucleases) gene-edited cell therapy for treating CD19-expressing hematologic cancers, initially acute lymphoblastic leukemia. In 2015, two infant leukemia patients who had failed previous therapy were treated using UCART19 in a first-in-man application. The cases were reported in Science Translational Medicine in January 2017. In November 2015, Servier exercised its option to acquire exclusive global rights to further develop and commercialize UCART19.

    Last mont, Cellectis was granted a therapeutic-focused European patent for the use of CRISPR technology, including Cas9 and Cpf1, for engineering T cells.

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