VCAR33ALLO (Allogeneic)

VCAR33ALLO is a CD33-directed CAR-T cell therapy made from allogeneic healthy donor-derived cells

We are developing a Chimeric Antigen Receptor T (CAR-T) cell product candidate, VCAR33ALLO, that targets the CD33 protein. Our ultimate vision is to develop a treatment system for AML where trem-cel is first administered to patients to remove CD33 from their healthy cells, followed by VCAR33ALLO administration to target and kill any remaining cancer cells.

Mylotarg™, an Antibody Drug Conjugate (ADC), is currently the only anti-CD33 therapy approved by the FDA. VCAR33ALLO uses a CAR construct developed by T-cell expert Dr. Terry Fry and licensed from the National Institutes of Health. We believe that VCAR33ALLO may be a better targeted therapy due to higher expected potency and longer expected persistence.

VCAR33ALLO uses cells derived from the same matched healthy donor who previously provided a stem cell transplant for the patient. This starting material has three key potential advantages: first, the cells are likely much more plentiful than cells sourced from the patient, who by this stage has likely received several lines of anti-cancer therapy; second, these cells are often more stem-like which could provide robust expansion and persistence in the patient; and lastly, these cells are exactly matched to the patient’s immune system having come from the same donor.

Vor Bio has constructed an in-house cell therapy manufacturing facility that is being used to manufacture VCAR33ALLO, using an abbreviated manufacturing process to maintain a stem-like cell phenotype alongside robust transduction of the CAR construct.

VCAR33ALLO Clinical Development

The FDA has cleared Vor Bio’s Investigational New Drug (IND) application for VCAR33ALLO, allowing for this therapy to be used in patients who have relapsed with AML after allogeneic hematopoietic cell transplantation (alloHCT). T cells from the patient’s original stem cell donor are used to manufacture VCAR33ALLO, thus generating CAR-T cells with potentially superior expansion capability and persistence compared with CAR-T cells manufactured from other cell sources.

VCAR33ALLO is being studied in the VBP301 clinical trial, which runs in two phases: the first phase, which is expected to enroll approximately 24-36 patients, is designed to determine the maximum tolerated dose of VCAR33ALLO using a 3+3 trial design; the second phase, which can enroll up to 60 additional patients, is an expansion phase designed to evaluate the rate of clinical response to treatment. Patients previously transplanted with trem-cel in the VBP101 study who have relapsed or refractory AML may be eligible for the VBP301 trial.

Related Publications

Next In Our Pipeline

VCAR33AUTO (Autologous)