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 Gaucher disease and Hunter syndrome. We are also advancing a program in Pompe disease.
“AVROBIO is comprised of pioneers who aim to bring personalized ex vivo lentiviral gene therapies to patients around the world. This purpose inspires us to work with urgency every day to move our programs forward.”
Steven Avruch, JD
“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.”
Meet our leadership team.
Explore careers at AVROBIO.
Learn more about our pipeline of investigational gene therapies.
CloseToday is MPS Awareness Day (MPS II is also known as Hunter syndrome) and we want to recognize the incredible work that patient advocacy groups do year-round to support the Hunter syndrome community.
Learn more about Hunter syndrome and the patient advocacy groups’ efforts here: