Vor Biopharma, an oncology company, announced an exclusive licensing agreement with the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), for intellectual property related to a clinical-stage anti-CD33 chimeric antigen receptor T-cell (CAR-T) therapy candidate. This CAR-T construct was devised by T-cell expert Dr Terry Fry during his tenure at the Pediatric Oncology Branch of the NCI, where he oversaw the development of this therapeutic candidate from bench to bedside; it is currently being evaluated in a multi-site Phase 1/2 clinical trial in children and young adults with relapsed or refractory acute myeloid leukaemia (AML).

“As a class, CAR-T cell therapies have had a major positive impact on the lives of certain patients with haematological malignancies. But because normal cells often express the same surface proteins as cancer cells, the utility and applicability of targeted therapies have been limited, in part, by on-target toxicity. This agreement provides Vor with access to a promising CD33 CAR-T that could potentially be administered either as bridge-to-transplant therapy for relapsed or refractory patients with AML, or following transplant with our lead developmental candidate VOR33, whereby the CAR-T may selectively target leukaemia cells while sparing normal myeloid cells,” said Christopher Slapak, Vor’s Chief Medical Officer.

Vor’s lead program VOR33, which is currently in pre-clinical development, consists of eHSCs that are engineered to provide AML patients with a donor-derived hematopoietic stem cell transplant that lacks the cell surface protein CD33, a clinically validated target for AML. The goal of removing this target is to make these eHSCs and their progeny treatment-resistant to anti-CD33 therapies. As such, Vor believes this CAR-T could be highly complementary to VOR33.

Other terms of the agreement have not been disclosed.