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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.

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AVROBIO has a preclinical research program for an investigational gene therapy for Gaucher disease type 3.

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).

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AVROBIO has a preclinical research program for mucopolysaccharidosis type II (MPS II), or Hunter syndrome. The program is being developed in collaboration with Brian Bigger, Ph.D., who has published favorable preclinical data in a mouse model of Hunter syndrome.

AVROBIO has a preclinical research program for a gene therapy for Pompe disease. We have shown a favorable preclinical safety and efficacy profile in a mouse model of Pompe disease.