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THE GREAT POTENTIAL TO CURE

Transformative gene therapies deliver new promise to patients.

Across a growing number of rare diseases, dramatic therapeutic outcomes
from gene therapies with well documented safety profiles
offer hope for a long sought-after curative benefit in a single dose.

Just as enzyme replacement therapy (ERT) revolutionized the past,
gene therapy will revolutionize the future.

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PROGRAMS

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

AVROBIO’s lentiviral gene therapy approach is proven, with a strong efficacy and safety track record.

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