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)
A 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 (α-Gal A). A phase 2 trial will be initiated in early 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)
The primary objective of the upcoming Phase 1/2 trial is to evaluate the safety and efficacy of AVR-RD-02, 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)
Pre-clinical experiments using our gene therapy platform and proprietary lysosomal targeting sequence demonstrated safety and efficacy in Pompe disease mice. Preclinical studies are underway to enable a future Phase 1/2 trial.
AVROBIO’s Gene Therapy for Cystinosis (AVR-RD-04)
The primary objective of the upcoming Phase 1/2 trial is to evaluate the safety and efficacy of using the patient’s own stem cells, which are genetically modified to express the CTNS protein, to treat the underlying disease.
Safety and efficacy have not been established nor have these investigational drug candidates been approved by the US Food and Drug Administration or any other regulatory agency.
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