At AVROBIO, we aim to halt or reverse disease with a single dose of personalized gene therapy. Our goal is 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.
Our investigational gene therapies start with the patient’s own stem cells. We are developing these ex vivo lentiviral gene therapy treatments for several serious lysosomal disorders, including clinical programs for Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.
We have spent years optimizing the elements of plato®, our proprietary platform designed to bring gene therapy to patients worldwide. plato has been developed with the goal of optimizing the safety, potency, and durability of AVROBIO's investigational lentiviral gene therapies.
To learn more about our approach, visit avrobio.com, and
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