Vice President, Clinical Development, Huntington's Disease Program Lead, Uniqure
Joseph Higgins, M.D., joined uniQure in March 2018 as Vice President of Clinical Development and is responsible for the early development of clinical gene therapy trials for neurological disorders.
Dr. Higgins serves as the lead for our Huntington's disease clinical program. The Company’s Investigational New Drug (IND) application for AMT-130 has been cleared by the FDA allowing uniQure to begin its planned dose-escalating, randomized and controlled Phase I/II clinical trial to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease.
Prior to joining uniQure in 2018, Dr. Higgins was responsible for all neurological diagnostic testing at Quest Diagnostics and was the federal CLIA-license holder for a specialty laboratory. He was formerly a Professor at Weill Cornell Medical College where he was the principal investigator on several private foundation and NIH research grants focused on clinical neurogenetics. He held senior leadership positions in genomic medicine at the federal and state governments including the National Institute of Health (NIH) and the New York State Department of Health (NYSDOH). At NIH he was part of the Human Genome Project and teams that initiated the first-in-human enzyme replacement and gene therapy clinical trials. He led the Human Genetic Initiative at the NYSDOH. He discovered the genes or loci for several diseases including Parkinson disease, essential tremor, ataxia, intellectual disability (ID), and metabolic disorders. He created a transgenic mouse model for proof-of-concept studies to treat non-syndromic ID.
He is a fellow of the American Academy of Neurology with board certifications in Pediatrics by the American Board of Pediatrics and Neurology with Special Qualification in Child Neurology by the American Board of Psychiatry and Neurology. He has authored more than 100 peer‐reviewed publications, book chapters, and reviews articles in the field of neurogenetics. He