Imagine Limitless Possibilities

Measuring our impact in lifetimes and life’s moments

Cecilia

Born with metachromatic leukodystrophy

Learn about families' experiences with MLD

Inspiration and Impact

HSC gene therapy has the potential to transform the lives of people living with severe and other genetic diseases. Read more about the stories and families that motivate us to achieve our mission.

Families

Pioneering Science

We’re building on decades of research to apply our HSC gene therapy platform to areas of significant medical need where we believe it may have distinct therapeutic advantages. Learn how our platform is designed to work.

Gene Therapy

Title of the wysi

The site uses cookies to provide you with a more responsive and personalized service. By using this site, you agree to our use of cookies as set out in our cookie policy.

Please read our privacy policy and cookie policy for more information on the cookies we use and how to delete or block the use of cookies.