Our
Pipeline
ONE PIPELINE for a better future
We are building a pipeline of compelling investigational therapies in indications where we believe gene therapy can have the greatest impact for people living with genetic disorders.
Our investigational therapies are being evaluated in clinical studies in Gaucher disease type 1 and cystinosis.
Discover more about how HSC gene therapy can have a head-to-toe impact
AVROBIO’s investigational gene therapy for Gaucher disease is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1.
The trial is now enrolling in Canada, Australia and the U.S. The trial is intended to recruit 8 to 16 individuals between the ages of 16 and 35 with Gaucher disease type 1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy.
AVROBIO’s investigational gene therapy for cystinosis is being studied in a Phase 1/2 investigational trial sponsored by the University of California, San Diego*. The first patient was dosed in October 2019. The single-arm trial is expected to enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis.
*Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).
Safety and efficacy have not been established. Our investigational therapies have not been approved by the U.S. Food and Drug Administration or any other regulatory agency.
Learn more about access to our clinical trials.
Learn more about our commitment to people living with genetic disorders.
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