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.
Scientific Advisory Board
Dr. Balkrishen Bhat, Ph.D.
- Vice President, Chemistry, Translate Bio
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
Peter Lansbury, Ph.D.
- Chief Scientific Officer, Lysosomal Therapeutics
- Professor of Neurology, Harvard Medical School
Beverly L. Davidson, Ph.D.
- Professor of Pathology & Laboratory Medicine The Children’s Hospital of Philadelphia
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
Elizabeth Fisher, Ph.D.
- Institute of Neurology, University College London, UK
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