Cellectis starts dosing patients in UCART123’s Phase I trial for BPDCN


Biopharmaceutical firm Cellectis has started dosing patients in a Phase I clinical trial of its universal chimeric antigen receptor T-cell (UCART) product candidate UCART123 to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN).

UCART123 is a gene-edited, T-cell investigational drug being developed to target CD123 antigen expressed on tumour cells of certain cancers such as BPDCN, an aggressive haematological malignancy.

Designed to assess the safety and efficacy of UCART123, the Phase I trial will include relapsed and refractory BPDCN patients in a front-line setting.

The first patient in the trial has been administered with the investigational candidate at MD Anderson Cancer Centre.

Cellectis chief medical officer Dr Loan Hoang-Sayag said: “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.

"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."

“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.”

In February, the US Food and Drug Administration (FDA) granted an investigational new drug (IND) approval for evaluation of UCART123 in patients with acute myeloid leukaemia (AML) and BPDCN.

UCART123 is claimed to be the first allogeneic, off-the-shelf, gene-edited, CAR T-cell CD123 targeting drug candidate to be evaluated in the US.

The UCART123 clinical programme is being led by MD Anderson Cancer Centre assistant professor Dr Naveen Pemmaraju, professor Marina Konopleva and professor Hagop Kantarjian from the Cancer Medicine division’s leukaemia department.