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Our mission is to develop transformative gene therapies that deliver new promise to patients. Across a growing number of rare diseases, dramatic therapeutic outcomes from gene therapies offer hope for a long sought-after curative benefit in a single dose. Just as enzyme replacement therapy (ERT) revolutionized the past, gene therapy may have the potential to revolutionize the future.
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PIPELINE

The aim of AVROBIO’s gene therapy programs is to deliver lasting and meaningful benefits for patients with rare genetic diseases. 

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Diseases

Our focus is on serious rare diseases, where a faulty gene results in a lack of an essential enzyme or protein.

CURE

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TECHNOLOGY

We are developing therapies using our gene therapy approach to transform a patient’s own cells into a drug product.

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