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

May 22 2023

AVROBIO Announces Agreement to Sell Cystinosis Gene Therapy Program for $87.5 Million

All-cash transaction, with full $87.5 million to be paid at closing Proceeds expected to extend cash runway into Q4 2024 CAMBRIDGE, Mass. --(BUSINESS WIRE)--May 22, 2023-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic

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May 18 2023

AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting

All patients remain off oral cysteamine up to 36 months post gene therapy Sustained engraftment and durable reduction in leukocyte cystine levels across all patients Received positive regulatory feedback from US and UK agencies CAMBRIDGE, Mass. --(BUSINESS WIRE)--May 18, 2023-- AVROBIO, Inc.

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May 11 2023

AVROBIO Reports First Quarter 2023 Financial Results and Provides Business Update

On track to initiate registrational global Phase 2/3 clinical trial for Gaucher disease type 3 (GD3) in second half 2023, subject to regulatory alignment Plan to provide clinical and regulatory updates on cystinosis program in conjunction with ASGCT annual meeting in May 2023 ; plan to initiate

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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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