Programs Header Scaled Aspect Ratio 1200 384

Programs

Putting our science into action

We are now working to bring our approach and the possibility of a cure to patients with high-risk Acute Myeloid Leukemia (AML), a disease that carries a poor prognosis even with aggressive therapies including Hematopoietic Stem Cell Transplant (HSCT), the current standard of care that has seen no significant change in decades.

Robust preclinical data supports the promise of our novel approach. Our engineered HSCs retain healthy stem cell function as demonstrated by the production of downstream progenitor cells with normal function. Furthermore, in preclinical models, transplantation with our engineered HSCs enables the successful clearing of AML cells with targeted therapies while preserving the normal blood cells.

Our research programs

VOR33

VOR33 is our lead eHSC product candidate created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target which is intended to protect them from a targeted therapy post-transplant.

VCAR33 programs

The VCAR33 programs are chimeric antigen receptor T (CAR-T) cell therapy candidates designed to target CD33, a clinically-validated surface target for AML.

VCAR33ALLO (Allogeneic)

VCAR33ALLO uses allogeneic healthy donor-derived cells.

VCAR33AUTO (Autologous)

VCAR33AUTO uses autologous cells from each patient.

Treatment systems

VOR33 + VCAR33 treatment system

VOR33 + VCAR33 Treatment System is a novel and comprehensive approach that has the potential to transform clinical outcomes and establish a new standard of care for patients suffering with AML.

VOR33-CLL1 + VCAR33-CLL1 treatment system

This Treatment System would remove multiple surface targets in eHSCs and use a multi-specific CAR-T.

Discovery platform

We are conducting ongoing discovery efforts in commonly transplanted hematologic malignancies.

Manufacturing

We are investing in our own manufacturing facilities and have designed a highly efficient process for manufacturing our eHSCs
and CAR-Ts.