Rate My Professor Ian Alexander

Professor Ian Alexander, BMedSci, MBBS (Hons), PhD, FRACP (Paediatrics), HGSA Clinical Geneticist, is Professor of Paediatrics and Molecular Medicine in the Faculty of Medicine and Health at the University of Sydney. He is Head of the Gene Therapy Research Unit, a joint initiative of Children’s Medical Research Institute and Kids Research at The Children’s Hospital at Westmead, which he founded over 25 years ago. He also serves as Director of Laboratory Research and Senior Staff Specialist at The Children’s Hospital at Westmead within the Sydney Children’s Hospitals Network. Involved in gene therapy research since 1992, he has played a pivotal role in establishing Australia’s first Good Manufacturing Practice viral vector facility and bringing advanced international paediatric gene therapy clinical trials to the country, including for spinal muscular atrophy.

Professor Alexander’s research specializations centre on gene therapy for genetic disorders of childhood, particularly liver diseases, employing genetically engineered viral vectors such as adeno-associated virus to repair or replace faulty genes. His team achieved Australia’s first gene therapy clinical trial for a genetic disease and has contributed significantly to understanding immune responses to gene therapeutics and advancing genome editing technologies. With over 290 peer-reviewed publications and more than 12,000 citations, his work has had substantial impact on the field. He is a Fellow of the Australian Academy of Health and Medical Sciences (FAHMS), recipient of the 2022 Peter Wills Medal from Research Australia for outstanding contributions to health and medical research, and has received prestigious NHMRC grants, including a top-ranked Ideas Grant. Professor Alexander contributes to clinical trial evaluations through the Kids Advanced Therapeutics program and fosters national collaboration for rare disease therapies.