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 Executive Officer
and President
Chief Medical Officer
Chief Commercial Officer
Chief Scientific Officer
Chief Technology Officer
Chief People Officer
Chief Legal Officer
and Corporate Secretary
Chief Financial Officer
Managing Member, Fairmount
Funds Management LLC
Director of Research, Fairmount
Funds Management LLC
Medical Oncologist and former Pharma-Biotech Executive
Chief Executive Officer,
Willow Advisors
Pharma-Biotech Executive
Pharma-Biotech Executive
Chief Executive Officer and President of Cogent Biosciences
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
NYU Langone Health
MD Anderson Cancer Center
Chief Medical Officer,
Flatiron Health
Massachusetts General
Hospital Cancer Center
Laura and Isaac Perlmutter Cancer Center