Avrobio, a Cambridge, Mass.-based clinical-stage biotechnology company developing gene therapies for rare diseases, completed a $60m Series B financing.
The round was co-led by Cormorant Asset Management and Surveyor Capital (a Citadel company), with participation from Aisling, Brace Pharma Capital, Eventide Asset Management and Morningside, and with existing investors Atlas Venture, SV Health Investors and Clarus Ventures.
Led by Geoff MacKay, President and CEO, Avrobio is a clinical-stage company focused on the development of its gene therapy candidate, AVR‑RD‑01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease.
The company – which also has offices in Toronto, ON – intends to use the proceeds to advance multiple gene therapies from its proprietary lentiviral platform, including the lead gene therapy, AVR‑RD‑01, currently in Phase 1 for Fabry disease, as well as three additional gene therapies for other lysosomal storage disorders, Gaucher disease, cystinosis and Pompe disease.
In advancing the gene therapy pipeline, Avrobio plans to initiate a Phase 2 clinical trial this year with AVR-RD-01 in Fabry disease, based on 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 plans to initiate clinical development of two other gene therapy candidates for cystinosis and Gaucher disease by mid-2019.