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 ongoing Phase 1 trial is currently enrolling patients in Canada. 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 enzyme α-galactosidase A (AGA). We expect to begin enrollment in a concurrent Phase 2 trial in mid-2018; the primary objective of that trial is to assess the safety and efficacy of AVR-RD-01.

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

We plan to initiate a Phase 1/2 trial to evaluate the safety and efficacy of AVR-RD-02 in patients with Type I Gaucher Disease. In this planned trial, the patient’s own genetically modified stem cells that will 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 safety and efficacy in Pompe disease mice.

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

In a planned academic sponsored Phase 1/2 trial, we expect the first patient will be dosed in 2019. The patient’s own stem cells, which are genetically modified to express the CTNS protein, are used to treat the underlying disease.

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