Patients & Families

Watch to learn more about our approach:

We seek to make healthy cells invisible to targeted therapies

For some types of AML, a stem cell transplant is the only curative option. However, in some cases of AML, there is a high risk of relapse even after a stem cell transplant. A major challenge for AML treatment after transplant is that many of the current therapies that are intended to kill the cancer cells can also target and destroy healthy blood cells. Our approach is based on a simple idea: make the stem cells 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 stem cell transplant 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.

Treatment process

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

HSCs Collected

HSCs collected from matched healthy donor

genome engineering

Genome engineering is used to remove desired surface targets

Vor eHSC transplant

Patient receives Vor 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

The data from Vor Bio’s preclinical studies clearly indicate that when combined with our modified stem cells, AML-directed therapies eradicate the cancer cells while the normal healthy blood cells continue to thrive and function as normal.

Based on the strength of the data from our preclinical studies, VOR33 has been granted Orphan Drug designation and Fast Track designation from the FDA and a Phase 1/2a clinical trial called VBP101 is now open and actively enrolling patients undergoing a transplant for AML with a high relapse risk. Vor Bio is working with clinical trial investigators at top transplant centers in the U.S. and Canada with close oversight from the U.S. Food & Drug Administration (FDA) and Health Canada, as well as an independent data safety monitoring committee. Together, these groups provide rigorous supervision to ensure patient and donor safety.

Learn more about our Clinical Trial:

Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML

If you have any questions, please contact our Clinical Team:

Patient Organizations:

Learn more from organizations that are working on behalf of patients with Blood Cancers:

Expanded access policy:

Expanded access, sometimes called “compassionate use”, provides a pathway for patients to gain access to investigational treatments for serious diseases or conditions. Expanded access refers to the use of an investigational therapy (one that has not been approved by the U.S. Food & Drug Administration (FDA) or other applicable regulatory authorities) outside of a clinical trial that is conducted under an Investigational New Drug (IND) application.

When considering expanded access, it is important to consider guidelines from the U.S. FDA as well as guidelines from regulatory agencies outside of the U.S. According to the FDA, expanded access may be appropriate when all of the following apply:

  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
  • There is no comparable or satisfactory alternative therapy to treat the disease or condition;
  • Patient enrollment in a clinical trial is not possible;
  • Potential patient benefit based on available safety and efficacy information justifies the potential risks of treatment;
  • Providing the investigational medical product will not interfere with, or delay, investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

Currently, Vor does not offer an expanded access program. Our goal is to provide patients with access to the engineered hematopoietic stem cell therapy programs that we are developing as safely and quickly as possible. We believe the most appropriate way to achieve this goal is through participation in our clinical trials. Vor may revise this policy at any time.

If you have questions about Vor’s expanded access policy, please contact We plan to respond within five (5) business days of receiving your questions.