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About 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.

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Events & Presentations

Our News Latest press releases

May 14, 2020

AVROBIO Presents New Preclinical Data on Lentiviral Gene Therapy Program for Pompe Disease at ASGCT 2020

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.

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May 13, 2020

AVROBIO Reports Updated Clinical Data from Investigational Gene Therapy Programs for Fabry Disease and Cystinosis

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

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May 07, 2020

AVROBIO Reports 1Q 2020 Financial Results and Provides Business Update

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

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