Vor Bio is developing a proprietary platform built on Hematopoietic Stem Cell (HSC) biology, genome engineering, and Chimeric Antigen Receptor T (CAR-T) cells that has the potential to protect HSCs from targeted therapeutics, enabling the use of modalities such as Antibody Drug Conjugates (ADCs) and CAR-Ts without risk of graft failure or cytopenia.
Our scientists and engineers are developing a proprietary platform that genetically modifies healthy donor HSCs to remove select cell surface targets, making these HSCs and their progeny resistant to targeted therapies. This would enable targeted therapies to selectively destroy cancer cells while passing over healthy cells. Limiting on-target toxicity previously associated with targeted therapies may allow their use soon after Hematopoietic Stem Cell Transplant (HSCT), enabling new treatment opportunities for these patients. By combining our engineered HSCs with targeted therapies—including CAR-Ts, bispecific antibodies, and ADCs—we have the potential to completely replace the traditional standard of care for blood cancers such as AML.
Preclinical data supports the viability of this new approach, demonstrating cellular protection without compromising cell populations or function. We are committed to sharing with the scientific community the data that underscores our approach. In partnership with leading transplant centers across North America, we are engaging in a first in-human clinical trial for this breakthrough approach.
Gene Editing and Immunotherapeutic Targeting of ADGRE2/EMR2 to Enable Combinatorial Targeting in Acute Myeloid Leukemia (AML)
Initial First-In-Human Results: CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplantation (HCT) and Tolerate Post-HCT Gemtuzumab Ozogamicin (GO) without Cytopenias
Translocation Detection and Quantification Workflow to Characterize CRISPR-Cas Multiplex Edited Hematopoietic Stem and Progenitor Cells (HSPCs)
Initial First-In-Human Results: CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplant (HCT) and Tolerate Post-HCT Gemtuzumab Ozogamicin (GO) without Cytopenias
Leveraging Human Genetics To Advance Cell Therapies For Treatment Of Blood Cancers
A machine learning approach incorporating germline information improves genotyping of CRISPR-Cas9 gene editing events at single cell resolution
Efficient Multiplex Gene Editing of CD33 and CLL-1 in Human Hematopoietic Stem Cells Enables the Potential of Next-Generation Transplants for AML Treatment
Functional Validation of Single Domain Antibody-Derived CD33 Specific CAR-T Cells for the Treatment of Acute Myeloid Leukemia
Multiplex deletion of myeloid antigens by base editing in human hematopoietic stem and progenitor cells (HSPCs) enables potential for next generation transplant for acute myeloid leukemia (AML) treatment
