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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 clinicaltrials@vorbio.com. We plan to respond within five (5) business days of receiving your questions.