Adj. Associate Professor Paula Bray is the Network Director of Research for the Sydney Children’s Hospitals Network. Paula completed her PhD and post-doctoral studies in measurement science and improving clinical care through development and implementation of international clinical practice guidelines for Duchenne muscular dystrophy and Charcot-Marie-Tooth disease. Paula is committed to advancing healthcare delivery through unlocking value by implementing research-enabled care. She works closely with research leaders, clinicians and partners to achieve national and international research impact that works to solve the biggest challenges facing children’s health.

Associate Professor Lisowski is a mid-career researcher with research interest is in genetic engineering of viral vectors, vector manufacturing and genome engineering for use in gene therapy approaches. He received his PhD from Weill Cornell University in New York for studies related to the development of lentiviral vectors for the treatment of β-thalassemia and completed postdoctoral training at Stanford University School of Medicine where he mastered design, production, and functional validation of novel bioengineered vectors based on adeno-associated virus (AAV). He developed the first bioengineered rAAV to enter clinical evaluation. In 2012 A/Prof. Lisowski was recruited by the Salk Institute for Biological Studies to oversee their Gene Transfer, Targeting and Therapeutic (GT3) facility. Based on his growing international reputation, in 2015 he was recruited by the University of Sydney / Children’s Medical Research Institute (CMRI) to establish his independent research team, Translational Vectorology Research Unit (TVRU) and with direct financial support from Luminesce Alliance to also establish Vector and Genome Engineering Facility (VGEF). His research concentrates on the studies of AAV biology and vectorology, and development of novel bioengineered AAV variants for clinical applications. His team has extensive expertise in the development and validation of in vitro, ex vivo and in vivo preclinical models of human disease, including the use of primary patient derived cells and organs and patient derived organoids. In addition, his group specializes also in development and improvement of viral vector manufacturing technologies, including upstream production and downstream purification, with special interest in clinical vector manufacturing. His team is leading a number of gene therapy programs that aim to develop novel advanced therapeutics for paediatric genetic disorders affecting the CNS, lungs, liver and other organs. Directly aligned with his mission to establish Australia as the global leader in gene therapies, together with the NSW Government and key decision makers at the Westmead Research Precinct, A/Prof. Lisowski is building a dedicated clinical grade vector manufacturing facility. This facility is a critical component that will enabling the development and clinical implementation of Australian gene therapies for paediatric and adult disorders, bringing the benefits of cutting edge science to the patients.

Adam Jaffe is the John Beveridge Professor and head of Paediatrics and Child Health at UNSW, Sydney, head of the Randwick Clinical Campus and a Respiratory Consultant at Sydney Children’s Hospital Randwick. Jaffe has expertise in rare diseases and chairs the Scientific and Medical Advisory Committee of Rare Voices Australia. He contributed to the writing of the National Strategic Action Plan for Rare Diseases and the National Recommendations for Rare Disease Health Care. Jaffe is also on the Rare Diseases International World Health Assembly resolution taskforce and the International Rare Disease Research Consortium n of 1 taskforce. Jaffe established the molecular and integrative precision medicine centre for cystic fibrosis at UNSW Sydney.

Dr Aung-Htut graduated with a PhD in Biochemistry and Molecular Genetics from the University of New South Wales (UNSW). Prior to her position as a senior research fellow (Co-Head of Molecular Therapy Laboratory) at the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT), Murdoch University, she held postdoctoral positions at the UNSW and Centre for Cancer Biology, University of South Australia working on cellular aging and transcriptional regulation of programmed cell death, respectively. Her current research focuses on designing and evaluating therapeutic antisense oligonucleotides for various diseases, including multiple sclerosis, juvenile-onset Parkinson’s, and Pompe disease. Dr. Aung-Htut aims to broaden the application of these therapeutic oligonucleotides to treat both rare genetic disorders and cardiovascular disease, the leading cause of death. Currently, Dr Aung-Htut and colleagues are driving towards innovative precision therapies for children with rare diseases, specifically n-of-1 approaches.

Since 2017 Sarah has Chaired the Sydney Children’s Hospital Network (SCHN) Human Research Ethics Committee (HREC), a Lead HREC under the National Mutual Acceptance Scheme and the NSW Health’s Statewide Early Phase Clinical Trial HREC for paediatrics. Sarah is an Associate Professor, Children's Hospital Westmead Clinical School, University of Sydney. Her research focuses on developing evidence-based interventions to prevent and treat childhood obesity and diabetes and she is currently on the scientific advisory panel of the EDIT Collaboration which brings together clinicians, researchers and individuals with lived experience from around the world to improve treatment for people affected by obesity and eating disorders.

Ms. Kris Pierce (MHealthSc (HealthProm&HealthEd), MWellness, Nursing) is a distinguished leader in consumer involvement and advocacy within the healthcare sector. As the Director of Consumer Involvement at Child Unlimited, UNSW, she has developed and implemented consumer engagement frameworks and led patient-centred initiatives. She has co-founded and leads SCN2A Australia and has contributed to the International League Against Epilepsy (ILAE), Global Genes, and The Epilepsy Foundation. Recognised through her induction into the Victorian Women’s Honour Roll and a nomination for the Children’s Healthcare Australasia Medal of Distinction, she continues to enhance patient and family engagement, ensuring consumer voices are integral to healthcare decision-making. Most recently, Kris was appointed to Medical Services Advisory Committee – Evaluation Sub-Committee.