Applying proven technologies in new ways to treat Acute Myeloid Leukemia (AML) and other blood cancers

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 shield healthy cells from targeted therapeutics, enabling the use of modalities such as Antibody Drug Conjugates (ADCs) and CAR-Ts which by themselves are highly toxic.

Vor Bio scientists and engineers are developing a proprietary platform for the treatment of blood cancers 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 and ADCs—we have the potential to completely replace the traditional standard of care for blood cancers such as AML and MDS.

In partnership with leading transplant centers across North America, we are conducting two clinical trials to evaluate this breakthrough approach. Our clinical data has demonstrated that our approach has the potential to shield patients’ healthy cells and enable targeted therapies post-transplant.