Gene Therapy Programs

The aim of AVROBIO’s gene therapy programs is to deliver lasting and meaningful benefits for patients with genetic diseases by restoring normal gene function and enzyme/protein production. 

AVROBIO’s Gene Therapy for Fabry Disease (AVR-RD-01)

An investigator-sponsored Phase 1 clinical trial is currently ongoing in Canada and is now fully enrolled with five patients dosed. The primary objective of this trial is to evaluate the safety and tolerability of infusing the patient’s own genetically modified stem cells that express the α-galactosidase A (AGA) enzyme. We are also enrolling in a separate company-sponsored Phase 2 trial at sites in Australia and Canada. The primary objective of the Phase 2 trial is to assess the safety and efficacy of AVR-RD-01. 

AVROBIO’s Gene Therapy for Gaucher Disease (AVR-RD-02)

We are now enrolling in a Phase 1/2 trial to evaluate the safety and efficacy of AVR-RD-02 in patients with Type I Gaucher Disease. In this investigational trial, the patient’s own stem cells are genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher Disease.

AVROBIO’s Gene Therapy for Pompe Disease (AVR-RD-03)

Preclinical experiments using our gene therapy platform and proprietary lysosomal targeting sequence demonstrated a favorable pre-clinical safety and efficacy profile in a mouse model of Pompe disease.

AVROBIO’s Gene Therapy for Cystinosis (AVR-RD-04)

A Phase 1/2 investigational trial sponsored by the University of California, San Diego is now enrolling patients. The patient’s own stem cells are genetically modified to express the CTNS protein with the aim of treating the underlying disease.

Safety and efficacy have not been established nor have any of our product candidates been approved by the U.S. Food and Drug Administration or any other regulatory agency.