Rare Disease Report

Biotech Advances Gene Therapies for Lysosomal Storage Diseases

FEBRUARY 01, 2018
Mathew Shanley
Clinical-stage biotechnology company AVROBIO, Inc. announced this morning that it has completed a $60-million Series B financing, of which proceeds will be used to advance several gene therapies, including AVR-RD-01, currently in Phase 1 for Fabry disease.

Additionally, gene therapies for three other lysosomal storage diseases will be advanced, including potential treatment options for Gaucher disease, Pompe disease, and cystinosis.

Lysosomal storage diseases are a group of rare genetic disorders that result from defects in the cellular vesicles that degrade large molecules, or lysosomes. People with these conditions are susceptible to a buildup of lysosomes in the cells, leading to multi-organ damage. Each disease is associated with a specific enzyme and the corresponding molecule that accumulates in the body determines the symptoms.

The advancement of AVR-RD-01 is based on promising six-month clinical results in the ongoing Phase 1 trial, in which a single dose exhibited the ability to produce normal plasma activity of α-galactosidase A enzyme, the lysosomal enzyme genetically deficient in patients with Fabry disease.

“Gene therapy is a class of medicines that has made breakthroughs in the biotechnology landscape, and AVROBIO has carved out a strong position with its lentiviral platform focused on the significant therapeutic and market opportunities for gene therapies for lysosomal storage diseases,” said Bruce Booth, D.Phil., Partner at Atlas Venture and Chairman of AVROBIO’s Board of Directors in a press release.

In addition to the continued work for AVR-RD-01, the company has said that it has plans to initiate clinical development of two other gene therapy candidates for Gaucher disease and cystinosis by mid-2019.

“AVROBIO has made remarkable progress with its gene therapy platform, including a very compelling initial clinical response in the first patient with Fabry disease to receive the company’s lead gene therapy candidate,” said Booth. “We have great confidence in the high caliber management team, and the rigorous science and clinical programs behind the gene therapies for Fabry disease and a range of other lysosomal storage disorders.”

With these prospective therapies, the company is boasting its lentiviral vector technology, a gene therapy transfer system used to stabilize the addition of genes into the patient’s own cells. It was developed to provide perpetual genomic integration for a long-lasting – potentially life-long – curative benefit.

With AVR-RD-01 and the other gene therapy candidates in its pipeline, AVROBIO is poised to become the first company to enter the clinic with gene therapies for lysosomal storage disorders. The company has stated its intentions to apply its proprietary gene lentiviral gene therapy platform to a broad range of other diseases where systemic delivery of gene therapy could provide therapeutic benefit.

“We are delighted by this exceptional group of investors supporting our plans to build AVROBIO as a leader in the field of lentiviral gene therapy,” said Geoff MacKay, President and CEO of AVROBIO. “We are excited to continue to rapidly advance our clinical program for AVR-RD-01 in Fabry disease, while also moving two additional gene therapies from our pipeline into clinical trials. We are on the leading edge of applying gene therapy as a potential single-dose curative treatment for lysosomal storage disorders, and our vision is to use our lentiviral platform to have broader impact for patients by shifting the paradigm for a wide range of other diseases.”

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