Hemophilia gene therapy clinical trial FAQs

Whether you are considering participating in an investigational gene therapy clinical trial for hemophilia or are looking to learn more about them, you need to do your research, ask questions, and get information. To help you along the way, here are some frequently asked questions that people in the hemophilia community are asking about investigational gene therapy clinical trials for hemophilia. You may use these as a starting point for discussions with your doctor and healthcare team.

Come back often, as we are gathering more questions and topics to potentially add as the website expands. This will help you in your research so that you can be informed and feel empowered throughout your hemophilia journey.

"Investigational" is a term that refers to a potential treatment being studied in clinical trials, but isn’t approved by regulatory agencies, such as the Food and Drug Administration (FDA). In clinical trials, scientists are studying to see if the investigational therapy is safe and effective, how much of the medicine may be needed, and how it may be used for that disease. Please consult with your doctor if you want to know more about participating in a clinical trial.