Avrobio 'on track' for two additional gene therapy programs in 2019
In October 2018, AVROBIO received a no objection letter from Health Canada to the clinical trial application for GAU-201, its Phase 1/2 clinical trial of AVR-RD-02 in Gaucher disease. The company subsequently received two additional NOLs from Health Canada to CTA amendments that incorporate elements of the plato platform into the trial. Dosing is expected to begin in the second half of 2019. In December 2018, the FDA accepted the Investigational New Drug application for an investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis. This trial will be led by Stephanie Cherqui, PhD, Associate Professor of Pediatrics at the University of California, San Diego, and is expected to begin in the second half of 2019.