Pipeline Aspect Ratio 1200 384

Our Pipeline

We are striving to bring fundamental change to cancer treatment

Our pipeline demonstrates the strength and potential of our novel approach to MDS and AML treatment based upon technologies in genome engineering, engineered Hematopoietic Stem Cell (eHSC) biology, and CAR-T cells with two clinical trials currently enrolling patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS).

Current Pipeline

Description
Pre-Clinical
Clinical
Program / Trial
Modality
Indication
Discovery
IND Enabling
Phase 1/2
Phase 2/3

Trem-cel + Mylotarg / VBP101

Trem-cel is a shielded transplant in development for patients with AML, in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to enable targeted therapies such as Mylotarg and CD33-targeted CAR-T therapy post-transplant, while avoiding on-target toxicities.

Learn more about our VBP101 clinical trial.

VCAR33ALLO / VBP301

VCAR33ALLO uses allogeneic healthy donor-derived cells. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the 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.

Learn more about our VBP301 clinical trial.

Trem-cel + VCAR33 Treatment System

We believe that the combination of trem-cel followed by treatment with VCAR33ALLO, our in-house CD33-directed CAR-T program, which we refer to as the trem-cel + VCAR33 Treatment System, in the post-transplant setting has the potential to transform patient outcomes in AML and establish a new standard of care for patients that have limited treatment options.

VADC45

VADC45 is a CD45-directed ADC. CD45 is an excellent target for a variety of blood cancers with clinical proof-of-concept.

CD33-CLL1 Treatment System

We are pursuing our first multi-targeted CD33-CLL1 Treatment System comprising a CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy. These next-generation, multiplex-edited HSCs may enable a wide range of treatment options post-transplant, including the use of multi-specific CAR-T therapies. Our dual-specific CAR-T uses the potency of two targets to address the large unmet need of patients with relapsed/refractory AML.

Program/Trial

Trem-cel + Mylotarg / VBP101

Trem-cel is a shielded transplant in development for patients with AML, in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to enable targeted therapies such as Mylotarg and CD33-targeted CAR-T therapy post-transplant, while avoiding on-target toxicities.

Learn more about our VBP101 clinical trial.

Modality
Shielded CD33-deleted transplant + CD33-directed ADC
Indication
AML, MDS
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
Program/Trial

VCAR33ALLO / VBP301

VCAR33ALLO uses allogeneic healthy donor-derived cells. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the 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.

Learn more about our VBP301 clinical trial.

Modality
CD33-directed transplant donor CAR-T
Indication
AML Post-transplant
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
Program/Trial

Trem-cel + VCAR33 Treatment System

We believe that the combination of trem-cel followed by treatment with VCAR33ALLO, our in-house CD33-directed CAR-T program, which we refer to as the trem-cel + VCAR33 Treatment System, in the post-transplant setting has the potential to transform patient outcomes in AML and establish a new standard of care for patients that have limited treatment options.

Modality
Shielded CD33-deleted transplant +
CD33-directed transplant donor CAR-T
Indication
AML
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
Program/Trial

VADC45

VADC45 is a CD45-directed ADC. CD45 is an excellent target for a variety of blood cancers with clinical proof-of-concept.

Modality
CD45-directed ADC
Indication
AML, conditioning, immune reset
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
Program/Trial

CD33-CLL1 Treatment System

We are pursuing our first multi-targeted CD33-CLL1 Treatment System comprising a CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy. These next-generation, multiplex-edited HSCs may enable a wide range of treatment options post-transplant, including the use of multi-specific CAR-T therapies. Our dual-specific CAR-T uses the potency of two targets to address the large unmet need of patients with relapsed/refractory AML.

Modality
Multi-specific CAR-T
Indication
AML
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
Program/Trial

CD33-CLL1 Treatment System

We are pursuing our first multi-targeted CD33-CLL1 Treatment System comprising a CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy. These next-generation, multiplex-edited HSCs may enable a wide range of treatment options post-transplant, including the use of multi-specific CAR-T therapies. Our dual-specific CAR-T uses the potency of two targets to address the large unmet need of patients with relapsed/refractory AML.

Modality
Multiplex-edited Shielded Transplant
Indication
AML
Preclinical
Clinical
Discovery
IND Enabling
Phase 1/2
Phase 2/3
AML: Acute Myeloid Leukemia
MDS: Myelodysplastic Syndrome
CAR-T: Chimeric Antigen Receptor T (cell)
ADC: Antibody Drug Conjugate
Platform

Our novel platform is based upon proven technologies in genome engineering, eHSC biology, and CAR-T cells.