Patients & Families

Expanded Access

REGENXBIO seeks to improve lives through the curative potential of gene therapy. 

We are committed to a rigorous clinical development process to evaluate the safety and efficacy of our investigational gene therapy candidates through clinical trials, while minimizing safety risks to patients.

We recognize that there are situations when a patient with a serious or life-threatening condition will not qualify for a REGENXBIO-sponsored clinical trial and may seek access to an investigational medicine outside of a clinical trial setting, also known as expanded access, treatment use or compassionate use.

At this time, REGENXBIO is focused on conducting clinical trials with the goal of achieving regulatory approval to make these therapies broadly available to patients.  For this reason, we are not accepting applications for expanded access.

Patients and families with questions can contact REGENXBIO’s Patient Advocacy team at Physicians with questions can contact our Medical Affairs team at

Further information on available REGENXBIO clinical trials can be found at our website,, or at the U.S. Government’s website for clinical trials, REGENXBIO reserves the right to revise this policy at any time, in accordance with the 21st Century Cures Act.