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 18 2022

AVROBIO Announces Preclinical Gene Therapy Data for Pompe Disease at American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Genetically modified hematopoietic stem cells lead to supraphysiological levels of therapeutic protein in a mouse model of Pompe disease sustained at eight months after administration Substantial reduction in glycogen observed across cardiac and skeletal muscles, as well as CNS Data support plans

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May 17 2022

AVROBIO Reports Positive Data From Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis, Including New Interim Data on Neurocognitive Measures

New early data show key visual motor integration, visual perception and motor coordination measures impacted by cystinosis stabilized or improved post gene therapy Systemic reach of AVR-RD-04 observed across multiple other measures All five dosed patients remain off oral cysteamine post gene

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May 16 2022

AVROBIO to Present at the H.C. Wainwright Global Investment Conference

CAMBRIDGE, Mass. --(BUSINESS WIRE)--May 16, 2022-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced members of its senior management team will be presenting virtually at the H.C.

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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

90+

years of cell and gene therapy experience among our leadership team

ONE
GOAL:

to give individuals with genetic disease freedom for life

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