23 Feb AVROBIO
Geoff MacKay, President and CEO
At AVROBIO we strive to give people with lysosomal disorders freedom for life through gene therapies designed to halt or reverse disease with a single dose. 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. The life-limiting symptoms patients with these disorders experience are caused by the lack of a crucial functional protein. Our personalized approach to gene therapy starts with inserting a therapeutic gene in the patient’s own stem cells to enable production of the protein they lack. Over time, these cells are expected to multiply and produce daughter cells. Each new generation of daughter cells is expected to contain the therapeutic gene. This approach holds the potential for the all-important protein to be durably expressed throughout the patient’s body, including the brain, delivering freedom for life.