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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 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.


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

Efficient knockout of both CD33 and CLL-1 by multiplex genome editing of human hematopoietic stem cells enhances the potential of next- generation transplants for acute myeloid leukemia (AML) treatment

TransACT enhances detection and characterization of translocation events from high-throughput sequencing data at base-pair resolution for gene editing products

Multiplex Deletion of Myeloid Antigens CD33 and CLL-1 by CRISPR/Cas9 in Human Hematopoietic Stem Cells Highlights the Potential of Next-Generation Transplantation for AML Treatment

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