WHAT IF ONE
GENE

Can change your entire world?

At AVROBIO, we aim to halt or reverse disease with a single dose of gene therapy. Our goal is simple and powerful: We aim to enable individuals with genetic disease to live longer — free from disease progression, free from painful and debilitating symptoms and free from the burden of chronic treatment regimens.

We're striving to deliver freedom for life

A highly personalized therapy

OUR FOCUSA highly personalized therapy

Our investigational gene therapies start with the patient’s own stem cells. We are developing these treatments for several serious lysosomal disorders.

Learn about our areas of focus.

Solving the challenges of gene therapy

OUR APPROACHSolving the challenges of gene therapy

We've spent years developing a state-of-the-art platform to optimize gene therapies. We call it plato®.

Get to know plato®.

OUR VIEWS The latest on gene therapy from our expert team

Read our insights

OUR NEWS Our latest press releases

Mar 1 2021

AVROBIO Receives Orphan Drug Designation from the European Commission for AVR‑RD‑04, an Investigational Gene Therapy for Cystinosis

AVR-RD-04 is the third AVROBIO investigational gene therapy to receive orphan drug designation CAMBRIDGE, Mass. --(BUSINESS WIRE)--Mar. 1, 2021-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today

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Feb 25 2021

AVROBIO to Present at Three Upcoming Investor Conferences in March

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 25, 2021-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in three upcoming

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Feb 8 2021

AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in First Patient Dosed with plato® Gene Therapy Platform in Fabry Disease Phase 2 Trial

Consistent with the 87% reduction in the first evaluable kidney biopsy, there is complete clearance of Gb3 inclusions in second evaluable kidney biopsy in Fabry disease Phase 2 trial New Gaucher disease type 1 Phase 1/2 data show plasma chitotriosidase levels decreased 49% and toxic metabolite

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WE ARE AVROBIO Join our team!

We are all about innovation. Collaboration. Dedication. And a quality we call “smart speed” — moving thoughtfully but with urgency to bring pioneering therapies to individuals living with genetic disease.

Explore careers at AVROBIO

By the numbers

3

countries where we’re conducting clinical trials

6

disease indications in our pipeline

30

years of foundational gene therapy science among our management team

ONE
GOAL:

to give individuals with genetic disease freedom for life

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