Rare Daily Staff

Gene therapy developer Avrobio said that it has completed a $60 million series B financing, which will be used to advance multiple gene therapies in its pipeline including AVR-RD-01, an  early-stage experimental gene therapy for Fabry disease, a rare metabolic disorder.

Cormorant Asset Management and Surveyor Capital co-led the round, which included participation by new investors Aisling, Brace Pharma Capital, Eventide Asset Management, and Morningside, along with existing investors Atlas Venture, SV Health Investors, and Clarus Ventures.

Avrobio said it plans to initiate a phase 2 clinical trial this year with AVR-RD-01 in Fabry disease, based on promising initial six-month clinical results in the ongoing Phase 1 trial. In the first patient with Fabry disease in the phase 1 trial, treatment with a single dose of AVR-RD-01 resulted in normal plasma activity of α-galactosidase A enzyme, the lysosomal enzyme genetically deficient in patients with Fabry disease.

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

“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, partner at Atlas Venture and chairman of Avrobio’s board of directors. “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.

With AVR-RD-01 and its pipeline of gene therapy candidates, Avrobio is positioned to be the first company to advance into the clinic with gene therapies for lysosomal storage disorders, a class of rare genetic diseases in which patients have a defective gene that produces an enzyme or protein that serves a vital metabolic function.

The company has initially applied its lentiviral gene therapy platform to lysosomal storage disorders and will also apply the platform to a broad range of other diseases where systemic delivery of gene therapy may be therapeutically beneficial. The company said the lentiviral vector technology is a gene transfer system for stable addition of genes into the patient’s own cells, designed to offer permanent genomic integration for a durable and potentially life-long curative benefit for patients.

“We are on the leading edge of applying gene therapy as a potential single-dose curative treatment for lysosomal storage disorders,” said Geoff MacKay, president and CEO of Avrobio, “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.”

February 1, 2018
Photo: Geoff MacKay, president and CEO of Avrobio