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

Sep 20 2022

AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis

Second AVROBIO gene therapy program that has been granted rare pediatric disease designation by FDA AVR-RD-04 has previously received orphan drug designation from FDA and EMA CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 20, 2022-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy

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Sep 14 2022

AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) or Hunter Syndrome Clinical Trial Application (CTA) Accepted by U.K. Regulatory Agency to Initiate Pediatric Phase 1/2 Gene Therapy Trial

First in-human pediatric Phase 1/2 study to evaluate the safety and tolerability of first-in-class gene therapy targeting infants age ≥3 months and ≤12 months, diagnosed with nMPS-II University of Manchester ( U.K. ) expects to initiate clinical trial later this year U.S.

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Sep 8 2022

AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 8, 2022-- AVROBIO , Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the company has granted an aggregate of 58,000 restricted stock units (RSUs) to three new

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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

4

countries where we’re conducting clinical trials

5

disease indications in our pipeline

ONE
GOAL:

to give individuals with genetic disease freedom for life

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