We are developing a Chimeric Antigen Receptor T (CAR-T) cell product candidate, VCAR33, that targets the CD33 protein.
VCAR33 is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell therapy that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33, the CD33-targeted CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes.
Vor Bio operates an in-house cell therapy manufacturing facility that is being used to manufacture VCAR33, using an abbreviated manufacturing process to maintain a stem-like cell phenotype alongside robust transduction of the CAR construct.
The U.S. Food and Drug Administration has granted Fast Track designation and Orphan Drug Designation to VCAR33, the CD33-directed CAR-T.
VCAR33 Clinical Development
We are actively enrolling VBP301 (NCT05984199), a Phase 1/2, multicenter, open-label, first-in-human study of VCAR33 in patients with relapsed or refractory AML after standard-of-care transplant or a trem-cel transplant.
The trial is evaluating safety, as well as key outcome measures including incidence of graft-versus-host disease related to VCAR33, percentage of patients who achieve response and overall survival and progression-free survival post-VCAR33 infusion.
The first phase, which is expected to enroll approximately 12 patients, is designed to determine the maximum tolerated dose of VCAR33 using a 3+3 trial design; the second phase, which is expected to enroll up to 12 patients, is an expansion phase designed to evaluate the rate of clinical response to treatment. The trial is designed to test the hypothesis that a CD33-targeted CAR-T derived from a healthy donor can be safely administered to a patient with AML who has relapsed after transplant and that the CAR-T can demonstrate anti-leukemia activity.
