We know every day matters to patients and their families. That’s why we are innovating what is possible in gene therapy with the goal of delivering one-time treatments to patients living with common and rare diseases.
Awarded Washington Post's Top Work Places in 2021 & 2022
We believe that what we do matters – to patients, to their families and to their communities. We set a high bar for having a culture focused on patients, driven by results, and one that fosters collaboration.