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 type 1 and cystinosis. We are also advancing a program in Gaucher disease type 3, Pompe disease and Hunter syndrome.
“We take technology leadership seriously; it’s essential to advance not just our programs but the gene therapy field as a whole. We must continually examine the lentiviral gene therapy process and push innovation at every step.”
“Being proactive is the key to our success at AVROBIO. Early on, we saw the need to assemble an efficient gene therapy platform and manufacturing plan that could produce consistent quality at scale as we prepare to deliver our investigational therapy to patients and families worldwide. Our forward-thinking efforts have resulted in our unique proprietary platform, plato®.”
Kim Raineri, MBA
“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.”
“At AVROBIO, we are committed to continually seeking out new ways to optimize technology and improve outcomes for patients. We know it will take many different approaches to reach all the patients with different genetic diseases in need. It’s that urgency that keeps us going every day.”
Meet our leadership team.
Explore careers at AVROBIO.
Learn more about our pipeline of investigational gene therapies.