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.
The aim of AVROBIO’s gene therapy programs is to deliver lasting and meaningful benefits for patients with rare genetic diseases.
Our focus is on serious rare diseases, where a faulty gene results in a lack of an essential enzyme or protein.
AVROBIO’s lentiviral gene therapy approach is proven, with a strong efficacy and safety track record.
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