Imagine Limitless Possibilities

Measuring our impact in lifetimes and life’s moments


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.


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

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