Compassionate Use Statement

Passage Bio is dedicated to developing transformative gene therapies for people with severe life-threatening, neurologic disorders. We approach our work every day with great urgency and commitment to the patients and families who suffer from these devastating diseases for which there are few or no treatment options.


To ensure that we meet our responsibility to deliver safe and effective treatments to patients, we collaborate with scientists, physicians, patients, and health authorities worldwide to conduct thorough clinical trials. We believe that it is through clinical trials and regulatory approval that we can ultimately ensure safe and timely access to our medicines for as many patients as possible suffering from these devastating diseases.

For information about our clinical trials, please visit or contact our Patient Services team at You may also have a member of your medical care team contact us at


We understand that there may be circumstances under which patients or caregivers may request treatment with our investigative therapies before they have been approved by regulatory agencies. This is sometimes called “expanded access,” “right to try,” “compassionate use,” “early access,” or a “named patient program.” We use the term “compassionate use” to capture all of these.  

Participation in clinical trials is the most common way to access investigational therapies. Clinical trials ensure that the safety and efficacy of the investigational therapy are evaluated prior to submission for review and potential approval by regulatory authorities. Therefore, after careful consideration, Passage Bio, as a clinical-stage company, has decided that the best way for patients to access our investigational therapies is through clinical trial participation. As a result, we will not offer compassionate use at this time.

Any requests for compassionate use will be referred to study investigators to determine a patient’s eligibility for one of our ongoing clinical studies. If a patient does not qualify for one of our ongoing clinical studies, Passage Bio will direct such patients, caregivers and/or their treating physician to patient organizations and/or medical professionals who may be able to identify other clinical studies or approved treatment options. 

Once we have completed and analyzed data from our registrational trials, (i.e., a clinical trial in which the results will be used to seek regulatory approval), we will evaluate the initiation of a compassionate use program. Should changes be made to our compassionate use policy, we will update this website.

Passage Bio may revise this policy at any time as provided by the 21st Century Cures Act.