The clinical-stage biotechnology company Avrobio has announced the expansion of its rare disease gene therapy pipeline to treat Pompe disease. The gene therapy is the company’s third targeting lysosomal storage disorders, following the ongoing Phase 1 trial for Fabry disease and pre-Phase 1/2 trial for Gaucher disease.
Pompe disease is caused by an inherited deficiency of the lysosomal enzyme acid α-glucosidase (GAA) leading to the accumulation of glycogen in muscles. Build-up of glycogen causes progressive muscle weakness throughout the body and affects various body tissues including heart, skeletal muscles, liver and nervous system.
Avrobio’s investigational treatment aims to target the faulty GAA gene via a modification of the patient’s own stem cells. The modified cells will then be infused back into the patient via a one-time treatment and is expected to sustain a long-term supply of the endogenous enzyme. It is hoped that the treatment will be able to replace the current biweekly intravenous infusions of enzyme replacement therapy.
Preclinical experiments conducted in mice models of Pompe disease have demonstrated safety and efficacy of the company’s proprietary gene therapy. Preclinical studies are now underway to enable a Phase 1/2 trial.
Geoff MacKay, Avrobio’s President and CEO commented: “By combining our state-of-the-art gene therapy platform with our proprietary lysosomal targeting technology, AVROBIO is advancing a truly novel solution to treat patients with Pompe disease.”
Source: AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease. Press Release