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VOR - Model VOR33 (CD33) - Hematopoietic Cells
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.
Vor is initiating VBP101, a Phase 1/2 clinical trial in patients with CD33-positive acute myeloid leukemia (AML) who are at high risk of relapse. The primary goals of the trial are to evaluate tolerability and feasibility of the VOR33 stem cell transplant, with a focus on confirming that VOR33 can engraft normally. Following engraftment, patients will be eligible to be treated with Mylotarg, a CD33-directed antibody drug conjugate (ADC) therapy, in order to potentially prolong leukemia-free survival and provide evidence that VOR33 protects against the myelosuppression that typically accompanies treatment with Mylotarg.