Vor Bio is currently conducting a clinical trial in AML

The data from our 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, trem-cel (formerly 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.

Joining a clinical trial

Clinical trials are highly regulated by the US Food and Drug Administration and other governmental regulatory agencies for the safety of patients and for the validity of the data. Vor Bio cannot help you in any way get into a trial. We can, however, direct you to information about the trial and to the clinical investigators who are managing the trial at the various transplant centers.

Talk to your doctor about eligibility

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below.

For general information:

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 Bio 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 Bio may revise this policy at any time.

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