Gene therapy on the Children’s Campus – how we got there and where we are going



The Melbourne Children’s Campus is seeing an increase in the number of clinical trials of gene therapies, especially in vivo viral vector therapies. With some of these therapies now approved by the TGA, there is an expectation by families that these therapies will be available at the Children’s.

In July 2021, RCH treated its first patient with Zolgensma, a gene therapy for spinal muscular atrophy. Preparations for the administration of the therapy took 18 months of planning, with set-up activities spanning multiple departments. With more of these therapies on the horizon, it is vital that RCH be adequately prepared to adopt this technology.

This Grand Rounds will discuss the mechanism of gene therapies, the regulatory environment, and the management of patients. The presentation will also highlight resources that are available, and outline how the Melbourne Children’s Campus aims to become a world leader in these therapies.



Professor Martin Delatycki is the Medical Director of Victorian Clinical Genetics Services and Co-Director of the Bruce Lefroy Centre at the Murdoch Children’s Research Institute. He has research and clinical interest in neurogenetic conditions, in particular Friedreich ataxia, and genetic screening. Martin has been involved in the process of achieving gene therapy trial readiness for the Children’s Campus.

Dr Ian Woodcock is a paediatric neurologist at the Royal Children’s Hospital and an honorary fellow at the Murdoch Children’s Research Institute. Ian has extensive experience as an investigator in multiple large-scale international industry-sponsored drug trials, including in Duchenne and other muscular dystrophies, and in breakthrough drug trials in Spinal Muscular Atrophy.

Katherine Lieschke has worked with the Melbourne Children’s Trials Centre at MCRI since 2016, primarily working on ethics and governance submissions for commercially sponsored clinical trials. She completed a Masters of Bioethics in 2019. Katherine is also a qualified teacher. In 2020 Katherine took on the role of Gene Therapy Trials Readiness Coordinator. In this role, she has developed a strong understanding of the regulation of gene therapy trials and products in Australia, and the challenges for sites in setting themselves up for success in this area.

Professor Monique Ryan is Director of the RCH Neurology Department and has extensive experience in neurogenetic research. Her group has contributed to identification of more than 15 disease-causing genes and to development of numerous medical and genetic therapies, including the first gene therapy approved for a childhood neurological condition. She collaborates with industry, regulatory authorities, and patient groups, established the Australian Neuromuscular Disease Registry, and has written more than 200 peer-reviewed publications and a textbook of paediatric neuromuscular disorders.

Professor Andrew Davidson is a Senior Staff Anaesthetist at the RCH, head of anaesthesia research at MCRI and Medical Director of the Melbourne Children’s Trials Centre (MCTC). The MCTC has been established as a joint Campus initiative to facilitate both investigator-initiated trials and commercially sponsored trials across a broad range of disciplines and trial designs. He is also currently chair of the Clinical Trails Subcommittee of the Melbourne Academic Centre for Health and Chair of the Network of Paediatric Trails Centres across Australia and chair of the Australian and New Zealand College of Anaesthetists Clinical Trials Network. His current interest is in advanced trial designs and facilitating trials into new types of therapies.

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