Every day, we come to work focused and driven by the opportunity to deliver the next best-in-class therapy for patients with genetically defined diseases – to move beyond incremental improvements and solely treating symptoms, to address the real causes of disease. We are methodical, rational, and intentional in our approach to identify pragmatic solutions to complex health challenges with the goal of restoring health and allowing patients to live better, longer lives.
Our Mission
Our Vision
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Chief Scientific Officer
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Senior Vice President, Chemistry
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Senior Vice President, Enzymology & Structural Biology
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Senior Vice President, Translational Science
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Senior Vice President,
Clinical Development
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Senior Vice President,
Head of Regulatory and Pharmacovigilance
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Vice President, Program Leader
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Senior Vice President,
Medical Affairs and
Chief Patient Officer
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Vice President, Clinical Pharmacology and Translational Medicine
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Senior Vice President,
Head of Clinical Development Operations