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

Chief Scientific Officer

Senior Vice President, Chemistry

Vice President, Enzymology
and Structural Biology

Vice President, Pharmacology and Toxicology

Vice President, Clinical Development

Senior Vice President,
Head of Regulatory and Pharmacovigilance

Vice President, Program Leader

Senior Vice President,
Medical Affairs and
Chief Patient Officer

Vice President, Clinical Pharmacology and Translational Medicine

Senior Vice President,
Head of Clinical Development Operations