Patients & Families

Homology Medicines is focused on advancing our genetic medicines platform as one-time treatments and potential cures for patients with rare diseases

Expanded Access Policy

At Homology Medicines, we are committed to developing safe and effective genetic medicines that have the potential to transform the lives of patients suffering from rare diseases. We are committed to bringing these important and innovative products to patients, and we believe in collaborating with patients, families, patient advocacy organizations, physicians, researchers and regulatory authorities to accomplish this goal.

Evaluation of an investigational therapy in clinical trials, which are designed to gain an understanding of the safety and efficacy of the therapy in a specific population, is the optimal way to collect the information necessary to enable regulatory authority review and, ultimately, to bring a therapy to the entire patient community. Participation in a clinical trial is also the best way for an individual patient to access an investigational therapy for his/her disease. At Homology Medicines, our goal is to enroll and carry out robust, thoughtfully designed clinical trials as expeditiously as possible in order to support regulatory approvals and thereby provide the broadest possible access to patients who might benefit from the therapy.

We understand that patients and families may be interested in accessing Homology Medicines’ investigational therapies prior to regulatory approval and outside of the clinical trial setting through an expanded access program (sometimes referred to as “compassionate use” or “early access”). At this time, we do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval. We encourage awareness of and participation in our clinical trials and believe that participating in clinical trials is the best way for patients to access our investigational therapies prior to regulatory approval. Treating physicians, patients, and/or caregivers interested in learning more about Homology Medicines’ investigational gene therapies currently undergoing clinical trials can find more information on If you have additional questions, please speak with your physician or contact We anticipate acknowledging receipt of requests sent to this email within five business days.