A clinical-stage gene therapy company striving to halt or reverse
disease with a single dose.
We are AVROBIO
Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato™ gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.Sign up for email alerts
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AVROBIO’s optimized lentiviral vectors demonstrate significant glycogen reduction in the muscle and central nervous system of Pompe disease mouse model Investigational New Drug (IND)-enabling proof-of-concept study for AVR-RD-03 for Pompe disease currently underway CAMBRIDGE, Mass.
Sustained enzyme activity up to 22 months and consistent trends demonstrated across multiple other measures in first patient in Fabry disease Phase 2 clinical trial Data from first patient treated using plato TM gene therapy platform in Fabry disease trial show: 43-percent reduction in toxic
Three oral presentations accepted on new clinical and preclinical data at the American Society of Gene and Cell Therapy (ASGCT) 23rd annual meeting Completed two collaboration agreements to reinforce technological leadership in lentiviral gene therapy Raised $100 million in gross proceeds from