About Us
Our Values
Deliver transformative patient impact
A patient's freedom to live without the burden of disease is our North Star.
The impact we deliver doesn't just change a life; it changes a family, a future, and the boundaries of what is medically possible.
Lead with excellence
You don't need a title to lead with excellence; you need a high standard and the commitment to hold it.
Excellence is our commitment to deliberately doing the right things.
Succeed through teamwork
We’re proactive, lean on each other's expertise, and own outcomes together.
We champion one another across teams and functions because we know the team isn't just the people sitting next to you.
Executive Leadership
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Jian Irish, PhD, MBA
President and CEO
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Pamela Wapnick, MBA
CFO
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Matthew Wein, JD
General Counsel, Compliance Officer, Head of BD&L & Corporate Secretary
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Alan Brooks, PhD
SVP, Research
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Katalin Kauser, PhD, MD, Sc.D
SVP, Translational Biology
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Mark Leonard, PhD
SVP, Technical Operations
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Kapil Saxena, MD, MS
VP, Clinical Development
Board of Directors
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Willard Dere, MD
Professor Emeritus, Internal Medicine, University of Utah
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Eric Bjerkholt, MBA
CFO of Mirum Pharmaceuticals, Inc.
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Juergen Eckhardt, MD, MBA
EVP, Head of Business Development & Licensing (BD&L) at Bayer, and Head of Leaps by Bayer
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Laurence Reid, PhD
Former President and CEO of Decibel Therapeutics, Inc.
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Jian Irish, PhD, MBA
President and CEO
Scientific and Medical Advisors
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Ken Takeshita, MD
Chief Scientific and Medical Advisor: Former Global Head of Research & Development for Daiichi Sankyo and Former Head of Clinical Development, Kite Pharma (now part of Gilead Sciences, Inc.)
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Glenn Pierce, MD, PhD
Scientific and Medical Advisor: Physician-researcher, hemophilia patient advocate, Former VP, Medical of World Federation of Hemophilia and Former SVP, Hematology and Gene Therapy, Biogen
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Steven Pipe, MD
Scientific and Medical Advisor: Professor of Pediatrics and Pathology at University of Michigan, Ann Arbor, Michigan
Help us define the future of genetic medicine
If you’re looking to do groundbreaking work with an inspiring and inclusive team, join us.