349 Vor Bbiopharma 2022.10.05 Genevieve De Manio Photography Scaled Aspect Ratio 1200 384

Our Investigational AML Treatment Approach

A new and transformative approach to Acute Myeloid Leukemia therapy

For some types of Acute Myeloid Leukemia (AML), a Hematopoietic Cell Transplant (HCT) is the only potentially curative option.

However, in many cases, AML can relapse even after HCT. A major challenge for AML treatment after Hematopoeitic Cell Transplant (HCT) is that many of the current therapies that are intended to kill the cancer cells can also target and destroy healthy blood cells.

Our AML targeted therapy approach is based on an elegant idea: make the Hematopoietic Stem Cells (HSCs) resistant to cancer-directed therapies so that healthy blood cells are protected, but cancer cells can be directly targeted for destruction.

Healthy donor stem cells are first edited to remove a marker on the outside of the cells so that they can no longer be recognized by AML-directed therapies. Once you recover from your HCT with the modified healthy donor cells, you may now be able to receive a targeted therapy to kill any remaining cancer cells and to decrease the chance that the cancer can come back because your healthy cells are now “invisible” to the targeted therapy.

This approach has been safe and effective in our preclinical studies: when combined with our modified stem cells, AML-directed therapies eradicate the cancer cells while the healthy blood cells continue to thrive and function as normal.

Acute Myeloid Leukemia Targeted Therapy Treatment Process

The patient is prepared to receive a hematopoietic stem cell transplant and HSCs are collected from a matched, healthy donor.

Hsc Matched Donor

HSCs collected from matched healthy donor

Genome Engineering

Genome engineering is used to remove desired surface targets

Hsc Transplant

Patient receives Vor Bio eHSC transplant

Once these eHSCs engraft, the patient may then receive a therapy that now attacks only the diseased cells that still have the target protein.


Healthy cells are invisible to targeted therapies

Target Attack

Learn more about our investigational treatment approach here:

Our clinical trials

Vor Bio is currently conducting two clinical trials for AML.