Our mission is to prevent repeat expansion disorders and ensure a future for our patients
We are developing a fundamentally different treatment for repeat expansion disorders — including Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias — by addressing the underlying cause of the genetic dysfunction that drives disease onset and progression.
To accomplish this goal, we’ve assembled a team with decades of expertise across drug discovery, genetics, clinical research and operations. We’re supported by a top-tier syndicate of investors who share our commitment to transforming the lives of patients and families affected by repeat expansion disorders.
Peter Bialek, Ph.D.
Director of Translational Sciences
Alan Buckler, Ph.D.
Jeffrey Cerio, Pharm.D., J.D
Principal Scientist, Pharmacology
Manager, Operations and Administration
Senior Vice President, Development Operations
Senior Scientist, Translational Sciences & Pharmacology
Satya Kuchimanchi, Ph.D.
Vice President, CMC
W. George Lai, Ph.D.
Vice President, DMPK & Toxicology
Senior Principal Scientist, Translational Biology
Senior Scientist, Molecular & Cell Biology
Senior Scientist, Bioanalytical & Quality Control
Senior Director, Program Management
Scientific Advisory Board
G. Bernhard Landwehrmeyer, M.D.
- Univ.-Prof. Dr. med., University of Ulm, Ulm, Germany
Jeffrey Carroll, Ph.D.
- Associate Professor, Behavioral Neuroscience Program and Department of Psychology, Western Washington University
- Co-founder and Editor-in-Chief, HDBuzz.net
Darren G. Monckton, Ph.D.
- Professor of Human Genetics, Institute of Molecular, Cell and Systems Biology, College of Medical, Veterinary and Life Sciences, University of Glasgow
Alexandra Durr, M.D., Ph.D.
- Coordinator of the National Center for Rare Diseases Neurogenetics
- Sorbonne Université, Institut du Cerveau et de la Moelle épinière (ICM), University Hospital Pitié-Salpêtrière, Paris, France
Dinah Sah, Ph.D.
- Former CSO, Voyager Therapeutics
- Former Vice President, Alnylam
- Former Associate Director, Biogen
Sarah J. Tabrizi, FRCP, Ph.D., FMedSci
- Director, Huntington’s Disease Centre, and Joint-Head, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology
- Principal Investigator, UK Dementia Research Institute at UCL
Andrew Fraley, Ph.D.
- Founder, Triplet Therapeutics, Inc.
- SVP Technology, Korro Bio
Vanessa Wheeler, Ph.D.
- Associate Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
Jim Gusella, Ph.D.
- Bullard Professor of Neurogenetics in the Department of Genetics, Harvard Medical School
- Director, Molecular Neurogenetics Unit, Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital
Ed Wild, FRCP, Ph.D.
- Associate Director, Huntington’s Disease Centre, University College London
- Professor of Neurology, University College London
Lesley Jones, Ph.D.
- Professor of Neurogenetics, Cardiff University
Gillian Bates, FRS FMedSci
- Professor of Molecular Neuroscience
- Co-Director of the Huntington’s Disease Centre
- Department of Neurodegenerative Disease and UK Dementia Research Institute
- Queen Square Institute of Neurology, UCL