Vor Biopharma products

VOR33 is our lead eHSC product candidate designed to replace the standard of care in transplant settings. Once the VOR33 cells have engrafted, we believe that patients can be treated with anti-CD33 therapies, such as Mylotarg or VCAR33, with limited on-target toxicity, leading to durable anti-tumor activity and potential cures. In preclinical studies, we have observed that the removal of CD33 provided robust protection of these healthy donor HSCs from the cytotoxic effects of CD33-directed companion therapeutics yet had no deleterious effects on the differentiation or function of hematopoietic cells.

Our first multi-targeted Treatment System is comprised of VOR33-CLL1 multiplex-edited eHSC therapy and VCAR33-CLL1 multi-specific CAR-T therapy. Knocking out CD33 and CLL-1 through gene editing offers a promising new approach to treating patients with AML using our novel eHSC platform. Our research demonstrates that multiplex genome editing of allogeneic hematopoietic stem cells may represent another exciting strategy to efficiently and safely edit multiple genes in blood stem cells, allowing the potential use of multi-targeted blood cancer therapies.

We believe VOR33 and VCAR33 could be highly synergistic as a Treatment System, potentially enabling prolonged remissions or cures in the post-transplant setting. We intend to investigate the VOR33/VCAR33 Treatment System, entailing VOR33 eHSC therapy followed by VCAR33 as a companion therapeutic, initially for transplant-eligible patients suffering from AML. We believe VCAR33 could be a potent anticancer therapy that, when combined with VOR33, could help obviate severe on-target myeloablative toxicities and unlock the efficacy potential of VCAR33. In addition, in this setting, VCAR33 T cells could be sourced from the same cell source as VOR33 (allogeneic cells), which may provide benefits such as a healthier, more abundant cell source alongside lower risk of host T cells attacking CAR-T cells, thereby potentially prolonging persistence. 

VCAR33^ALLO uses allogeneic healthy donor-derived cells. There has been an increasing appreciation for the value of cell phenotype in CAR-T approaches, and HLA-matched healthy donor cells are a potentially superior cell phenotype with improved persistence and in vivo expansion capability.

Licensed from the National Institutes of Health, VCAR33 is a CD33-directed chimeric antigen receptor T cell (CAR-T) therapy. A T cell therapy using the same CAR construct as VCAR33 is being studied in a multi-site Phase 1/2 clinical trial as an autologous monotherapy bridge-to-transplant for relapsed and/or refractory AML patients, sponsored by the National Marrow Donor Program (NMDP).