Toward the next generation of gene therapy
Imagine a world where the progression of debilitating chronic conditions can be stopped—with a single dose of genetic medicine. Where gene therapy can truly alter the course of a disease and change the course of people’s lives. It’s this vision that drives us forward.
That spurs us to keep pushing the boundaries of genetic medicine, toward what we know it can be.
Toward life-changing therapies, and brighter futures. Toward more.
Advancing the practice of genetic medicine
We’re leveraging the power of getting just the right gene expression, packaged and delivered to the body in just the right way, to develop transformational gene therapies for more diseases.
Targeting more conditions to impact more lives
Building on our success with a groundbreaking gene therapy candidate for Gaucher disease, we’re moving from rare diseases toward more widespread conditions, including forms of Parkinson’s and dementia, and even certain cardiovascular diseases.
