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Our Pipeline

We are striving to bring fundamental change to cancer treatment

Our pipeline demonstrates the strength and potential of our novel platform, with our lead clinical-stage program currently recruiting patients with relapsed/refractory AML.

Current Pipeline

Ind Enabling
Phase 1/2
Phase 2/3

Trem-cel (formerly VOR33)

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

VCAR33 (Allogeneic)

VCAR33ALLO uses allogeneic healthy donor-derived cells. There has been an increasing appreciation for the value of cell phenotype in CAR-T approaches, and HLA-matched healthy donor cells are a potentially superior cell phenotype with improved persistence and in vivo expansion capability.

VCAR33 (Autologous)

Licensed from the National Institutes of Health, VCAR33 is a CD33-directed chimeric antigen receptor T cell (CAR-T) therapy. A T cell therapy using the same CAR construct as VCAR33 is being studied in a multi-site Phase 1/2 clinical trial as an autologous monotherapy bridge-to-transplant for relapsed and/or refractory AML patients, sponsored by the National Marrow Donor Program (NMDP).

*A T cell therapy using the same chimeric antigen receptor construct as VCAR33ALLO is being studied in a Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (“NMDP”), and timing of data release is dependent on the investigators conducting the trial. Although we are not the sponsor of this trial, the NMDP has permitted us to cross-reference its IND for this trial in future IND applications that we may submit with the FDA. For more information regarding the NMDP trial, see “Risk Factors – We have not successfully tested our product candidates in clinical trials and any favorable preclinical results are not predictive of results that may be observed in clinical trials” in our Annual Report on Form 10-K for the year ended December 31, 2021 filed with the SEC and such other filings that we may make with the SEC from time to time.

Trem-cel + VCAR33

We believe trem-cel and VCAR33 could be highly synergistic as a Treatment System, potentially enabling prolonged remissions or cures in the post-transplant setting. We intend to investigate the trem-cel/VCAR33 Treatment System, entailing trem-cel eHSC therapy followed by VCAR33 as a companion therapeutic, initially for transplant-eligible patients suffering from AML. We believe VCAR33 could be a potent anticancer therapy that, when combined with trem-cel, could help obviate severe on-target myeloablative toxicities and unlock the efficacy potential of VCAR33. In addition, in this setting, VCAR33 T cells could be sourced from the same cell source as trem-cel (allogeneic cells), which may provide benefits such as a healthier, more abundant cell source alongside lower risk of host T cells attacking CAR-T cells, thereby potentially prolonging persistence. If our anticipated trials for the trem-cel/VCAR33 Treatment System are successful, we will have the potential to provide a single-company solution for patients suffering from certain hematological malignancies.


Our first multi-targeted Treatment System is comprised of CD33-CLL1 multiplex-edited eHSC therapy and CD33-CLL1 multi-specific CAR-T therapy. Knocking out CD33 and CLL-1 through gene editing offers a promising new approach to treating patients with AML using our novel eHSC platform. Our research demonstrates that multiplex genome editing of allogeneic hematopoietic stem cells may represent another exciting strategy to efficiently and safely edit multiple genes in blood stem cells, allowing the potential use of multi-targeted blood cancer therapies.

Discovery Platform
  • Leveraging our proprietary Vor Bio platform, we are exploring additional surface targets such as CD123, EMR2, and CD5 including multiplex genome engineering approaches where multiple surface targets are removed.
  • We are conducting ongoing discovery efforts in commonly transplanted hematologic malignancies.





AML: Acute Myeloid Leukemia
MDS: Myelodysplastic Syndrome
MPN: Myeloproliferative Neoplasm
CAR-T: Chimeric Antigen Receptor T (cell)
eHSC: engineered Hematopoietic Stem Cell
ADC: Antibody Drug Conjugate

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