ONE COMPANY with a vision to bring personalized gene therapy to the world.
We are 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.
Our goal is simple and powerful: 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.
Our initial focus is on lysosomal disorders, where the standard of care does not halt disease progression or adequately address many symptoms, particularly in the brain and nervous system. Our investigational therapies are currently being studied in clinical trials in Fabry disease, Gaucher disease and cystinosis. We are also advancing a program in Pompe disease.
“Gene therapy represents a new frontier of medicine with tremendous potential to overcome genetic disease. At AVROBIO, we are committed to developing safe and effective gene therapies across multiple indications to improve the lives of many.”
“We are on the cusp of a sea change in medicine thanks to the life-changing promise of cell and gene therapies. I know this optimism is shared by many who have been impacted by rare diseases or cancer, including my daughter.”
Deanna Peterson, MBA
“Great things can happen when you assemble a team that collaborates well and respects each other. We are fortunate to have built such a team at AVROBIO.”
Erik Ostrowski, MBA
“My life is gene therapy. Why? It’s an incredible scientific advance. Just in the past few years, we have seen that it can transform lives. I can’t think of a more exciting place to be than here, breaking new ground on this important frontier..”
Chris Mason, MD, PhD, FRCS, FMedSci