Cellectis’ UCART123 Administered to First Patient with BPDCN in Phase I Clinical Trial at MD Anderson Cancer Center

Published on August 17, 2017 in New York (N.Y.)

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UCART123 is First U.S. Gene Edited, Off-the-Shelf CAR T-Cell Program


August 17, 2017 – New York (N.Y.) – Cellectis (Alternext: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART), announced today that the first patient with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) has been dosed in Cellectis’ Phase I clinical study using the Company’s wholly controlled TALEN® gene edited product candidate UCART123 at the MD Anderson Cancer Center. UCART123 is the first allogeneic, “off-the-shelf” gene edited CAR T-cell product candidate targeting CD123 to be investigated in U.S. clinical trials.

The UCART123 clinical program for BPDCN is led by Dr. Naveen Pemmaraju, MD, Assistant Professor, Professor Marina Konopleva MD, PhD, and Professor Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine, at the MD Anderson Cancer Center.

The clinical trial will investigate the safety and efficacy of UCART123 in patients with BPDCN in the relapsed, refractory and front-line setting. BPDCN is a rare and aggressive hematological malignancy classified in the myeloid diseases among the acute leukemias that are derived from plasmacytoid dendritic cell precursors. It is a bone marrow disease that also often affects skin and lymph nodes.

Given its rarity and recent recognition as a distinct clinicopathological entity, no standardized therapeutic approach has been established for BPDCN, and the optimal therapy remains to be defined. Although transient responses are achieved by combination chemotherapy regimens that are used to treat acute leukemia or lymphoma, most patients relapse with the drug-resistant disease.

“We are eager to progress through clinical trials with UCART123, Cellectis’ wholly controlled gene-edited product candidate, next with the treatment of BPDCN, rare but aggressive entity,” said Dr. Loan Hoang-Sayag, Cellectis’ Chief Medical Officer. “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.”


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