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. 2021 Jun 3;137(22):3145-3148.
doi: 10.1182/blood.2020009759.

Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

Martin Wermke  1   2 , Sabrina Kraus  3 , Armin Ehninger  4 , Ralf C Bargou  5 , Maria-Elisabeth Goebeler  5 , Jan Moritz Middeke  1   2 , Carla Kreissig  6 , Malte von Bonin  1   2 , Jan Koedam  4 , Michael Pehl  4 , Martin Bornhäuser  1   2 , Hermann Einsele  3 , Gerhard Ehninger  4 , Marc Cartellieri  4
Affiliations

Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

Martin Wermke et al. Blood. .
No abstract available

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Conflict of interest statement

Conflict-of-interest disclosure: A.E., J.K., M.P., G.E., and M.C. are employed by and/or hold shares of GEMoaB GmbH. C.K. is employed by CMT Cellex Manufacturing Transports and Logistics GmbH. M.W. and H.E. received honoraria from GEMoaB GmbH for their advice related to this study. The remaining authors declare no competing financial interests.

Figures

Figure 1.
Figure 1.
First-in-human proof-of-concept for a rapidly switchable UniCAR targeting CD123 for treatment of AML. (A) Schematic presentation of the UniCAR platform. Autologous T cells are genetically engineered to express UniCAR, which does not recognize a surface protein, and hence, UniCAR-T are inactive under physiologic conditions. A soluble adapter consisting of the UniCAR epitope (UCE) linked to a single-chain fragment variable directed against CD123 (TM123) is required for antigen-specific redirection and activation of UniCAR-T. (B) Rapid recovery of leukocytes (WBC), neutrophils, and platelets in 3 treated AML patients after stop of TM123 infusion. Asterisks indicate timing of platelet transfusions. (C) UniCAR-T-cell numbers in peripheral blood (PB) over the course of treatment. Cell number was calculated from vector copy number determined by digital droplet polymerase chain reaction, mean and standard deviation are shown.
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