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Cellectis begins UCART123 clinical trial in BPDCN patients

Cellectis has administered its gene-edited CAR T-cell therapy UCART123 to the first patient with BPDCN, a rare and aggressive blood cancer, at MD Anderson Cancer Center in the U.S.

15 June 2026
Cellectis begins UCART123 clinical trial in BPDCN patients

Biopharmaceutical company Cellectis has initiated a Phase I clinical study in the United States using its product candidate UCART123. The first patient with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), a rare and aggressive blood cancer, received treatment at the MD Anderson Cancer Center in Texas.

UCART123 is a gene-edited CAR T-cell therapy targeting CD123. It is developed as an "off-the-shelf" product, meaning it can be used for multiple patients without individualized manufacturing, potentially reducing costs and wait times. This marks the first time the therapy is being evaluated in U.S. clinical trials.

BPDCN is a rare and aggressive hematological malignancy affecting the bone marrow and often the skin and lymph nodes. It is considered difficult to treat, with existing therapies like combination chemotherapy often yielding only transient responses before disease relapse.

The trial, led by physicians at MD Anderson, will assess the safety and efficacy of UCART123 in patients with front-line, relapsed, or refractory BPDCN. Cellectis aims to provide a new therapeutic option for this challenging cancer through gene editing.

Original source: cellectis.com