Expanded Access Policy

At Jaguar Gene Therapy, our mission is to accelerate breakthroughs in gene therapy for patients suffering from severe genetic diseases. We carry out this important work according to our core values of Integrity, Agility and Collaboration.

Expanded access, also known as compassionate use, is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.1

We understand the importance and need for expanded access programs in diseases with high unmet need. At this time, Jaguar Gene Therapy does not offer any expanded access programs, as our lead gene therapy candidate is in the early stages of clinical development with the aim of first evaluating safety and efficacy. Additionally, we are working to conduct preclinical studies to support advancement of our pipeline programs to the clinical stage.

We encourage individuals who are interested in our investigational gene therapies or pipeline to continue to follow our progress and if appropriate, talk with their doctor about participating in a clinical trial once they begin. Information about our future clinical trials, including eligibility criteria and locations will be made available at ClinicalTrials.gov at the appropriate time.

If we are able to offer expanded access in the future, we will update this policy. If you have further questions, please email eap@jaguargenetherapy.com.

  1. https://www.fda.gov/news-events/public-health-focus/expanded-access