Patients & Families

Expanded Access

REGENXBIO is seeking to improve lives through the curative potential of gene therapy. We actively engage with patients, caregivers and families to learn from their personal experiences in order to guide our work. Partnering with the communities we support helps us advance therapies that will meet their unique needs.

We are also committed to a rigorous clinical development process, to evaluate the safety and efficacy of investigational treatments through clinical trials, while minimizing safety risks to patients. If clinical trials establish that an investigational medicine is safe and effective, marketing approvals will be sought from regulatory health authorities with the goal of making these treatments broadly available to the patient communities.

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. While participation in clinical trials is the preferred way for patients to gain access to REGENXBIO investigational therapies, expanded access requests will be considered in limited situations according to available guidelines from regulatory authorities including, but not limited to, the following criteria:

  • The condition or disease being studied is serious, irreversibly disabling or immediately life-threatening and there are no acceptable alternative treatments for the patient.
  • The patient is not eligible for an ongoing or planned REGENXBIO-sponsored clinical trial of the investigational gene therapy product for their condition.
  • The request for expanded access has been made by a qualified and licensed physician with capabilities to monitor, manage and report side effects of the therapy as well as patient experience.
  • There is sufficient evidence of safety and pharmacologic effect for the investigational product, and the potential benefits to the patient outweigh the potential risks with the investigational product. 
  • There is adequate supply of the investigational product.
  • The patient is willing to travel to a REGENXBIO-approved facility with necessary expertise to administer a gene therapy investigational product and get the appropriate follow-up and monitoring post-therapy.
  • The patient can commit to long-term follow-up after gene therapy treatment (up to 5 years).
  • Providing expanded access to the investigational product is not expected to adversely interfere with or compromise the clinical development process.
  • The patient meets any other relevant medical criteria for expanded access to the investigational product, as determined by REGENXBIO.

Complete requests for expanded access will be evaluated in a fair, equitable manner; however, REGENXBIO cannot guarantee that the investigational gene therapy will be available to a specific patient. Any expanded access will also be subject to local laws or regulations, and the patient must provide informed consent. Approval of requests for expanded access must be obtained from the applicable regulatory health authority and Institutional Review Board or Ethics Committee.

Further information on available REGENXBIO clinical trials can be found at our website,, or at the U.S. Government’s website for clinical trials, Requests for information on expanded access to investigational products must be made by a licensed physician and may be submitted to It is anticipated that acknowledgement of any expanded access questions or requests will be made by REGENXBIO within five (5) business days of receipt. REGENXBIO reserves the right to revise this policy at any time, in accordance with the 21st Century Cures Act.