Patients & Families

Everything starts with our passion for the patient.

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We have been pursuing our mission—to deliver curative therapies that transform the lives of patients—since our beginnings. Today, we are focused on advancing our first two potentially transformative gene therapies for hemophilia B and Huntington’s disease.

Huntington’s disease

New hope for patients and families at risk for Huntington’s disease, a rare and fatal disorder of the central nervous system.

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Hemophilia Type B

A potentially curative, single-treatment gene therapy in late-stage development, partnering with global biotech leader CSL Behring.

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Patient stories

Transforming lives is at the heart of what we do. Our patients are everything to us—meet some of them here.

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Community resources

You are not alone. Browse here for information and support for patients and their families.

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