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The Science

Applying proven technologies in new ways

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.

By protecting healthy cells, Vor Bio is aiming to delay relapse post-transplant and potentially cure Acute Myeloid Leukemia (AML) and other blood cancers.

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 Cell Transplant (HCT), 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.


CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplant (HCT) and Tolerate Post-HCT GemtuzumabOzogamicin(GO) Without Cytopenias

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

Our Programs

Our novel platform is driving multiple programs in our pipeline

Our Programs

See how we apply the science behind our approach in our programs