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.
“At AVROBIO, we spent the past five years building a leading lysosomal disorder pipeline based on the knowledge that many debilitating rare diseases could benefit from a lentiviral gene therapy approach. Now, we’re focused on the next five years: progressing our leading programs out of the clinic and bringing a portfolio of important gene therapies to the patients and families in urgent need of a better solution.”
“To change the standard of care you need courage and a pioneering mindset. We’ve assembled a collaborative team of brave, bold thinkers who know their work has the power to truly transform lives.”
“Every second of every minute of every day counts and I don’t take that lightly. I couldn’t be prouder to lead AVROBIO’s science team. Gene therapy is no longer an idea on paper, it’s a reality, and that’s thrilling.”
Chris Mason, MD, PhD, FRCS, FMedSci
Meet our leadership team.
Explore careers at AVROBIO.
Learn more about our pipeline of investigational gene therapies.