Imagine Limitless Possibilities
Measuring our impact in lifetimes and life’s momentsInspiration 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.
FamiliesPioneering 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
“Coming out of #JPM2022 Orchard will ride this momentum as we continue to develop the potential of HSC Gene Therapy. We look forward to reaching new heights, looking to new horizons in 2022.”
“There is still work to do, and we are starting 2022 with the same passion and commitment to continue building a company focused on changing the treatment paradigm for patients with severe genetic diseases.” – Bobby Gaspar, CEO
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