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SESSION 3.1 - Functional Genomics and Precision Therapy: Cell And Gene Therapy Advancements: From Bench to Bedside

Tracks
Track 1
Thursday, November 7, 2024
2:00 PM - 3:30 PM
Leighton Hall, John Niland Scientia Building
Sponsored By:

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Prof Roger Reddel
Children’s Medical Research Institute

Chair

Biography

BSc (Med) MBBS PhD FRACP FAAHMS FAA. Roger has a long and distinguished career in oncology and medical research, including establishing the Cancer Research Unit at the Children’s Medical Research Institute (CMRI) more than 30 years ago, which he continues to lead. He has been the executive director of CMRI and the Sir Lorimer Dods Professor, at Sydney Medical School, University of Sydney, since 2007. Roger’s research focus is the immortality of cancer cells, an almost universal characteristic of cancers, with a particular focus on the role of chromosome ends (telomeres). The main aim of this work is to find new cancer treatments, but it is also contributing to understanding premature ageing in children and ageing in adults. With a keen interest in precision cancer medicine, big data and creating high-performing teams, Roger values the opportunities created by Luminesce Alliance for researchers to align their efforts for the benefit of children and their families. Roger has extensive experience of serving on medical advisory panels and has been elected a Fellow of the Australian Academy of Science and of the Australian Academy of Health and Medical Sciences. He has been awarded the Ramaciotti Medal for Excellence in Biomedical Research, the NSW Premier’s Award for Outstanding Cancer Researcher of the Year, and the Neil Hamilton Fairley Medal of the Royal Australasian College of Physicians. In 2021, he was awarded an Order of Australia. Directorships: Executive Director – Children’s Medical Research Institute (CMRI); Member of Board of Directors – Garvan Institute of Medical Research.
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A/Prof Anai Gonzalez-Cordero
Group Leader, Stem Cell Medicine And Manager, Stem Cell Facility
Children’s Medical Research Institute

Cell therapy pipeline, past, present, and future success

2:00 PM - 2:15 PM

Biography

Associate Professor Anai Gonzalez-Cordero is a Al & Val Rosenstrauss Fellow, and a Group Leader at Children’s Medical Research Institute (CMRI), The University of Sydney. Dr Gonzalez-Cordero is a leader in the field of stem cells and their differentiation into organoids with an emphasis on translational research to develop novel therapies for retinal genetic diseases. Having started her scientific career in the UK, she obtained a Wellcome Trust PhD in Stem Cell and Developmental Biology and continued her work at the Institute of Ophthalmology, University College London. In 2019, Dr Gonzalez-Cordero relocated to Australia to continue her research programme in stem cells and ophthalmology. Her work in the field has led to numerous awards, including the MetCalf Prize for Stem Cell Research in 2022, and high impact studies including proof-of-concept studies for stem cell-based cell therapy by transplantation of the light sensing cells, the photoreceptor cells, which once in the eye can rescue vison perception in blind animal models. At CMRI she also heads the Stem Cell and Organoid Facility proving iPSC lines and human organoid models for Australian researchers.
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Dr Shafagh Waters
UNSW Sydney

The world of avatar technology for cystic fibrosis and beyond

2:15 PM - 2:30 PM

Biography

Dr Shafagh Waters (BSc, MSc (Disc.), PhD) is a Scientia senior lecturer at UNSW and an honorary senior scientist at Sydney Children’s Hospital. A productive PhD (2012; ANU) and postdoctoral fellowships (2013-2016; UNSW) helped her secure international training fellowships in gene therapy (City of Hope USA) and organoid medicine (Lisboa, Portugal) establishing her independent lab in 2016. Dr Waters lead an NHMRC funded research program on adult-stem-cell biology for cystic fibrosis (CF) that is supported by 32 grants; 21 as CIA including international and national industry partnerships. Dr Waters is a founding member of NSW Non-Animal Technologies Network, has developed an Australian national biobank of stem-cell-derived airway and gut organoids, and has built a platform for high-throughput therapy-testing on patients organoids. She combines her unique strengths in organoid disease modelling, multi-omic molecular profiling and computational research with clinical data to improve individualised outcomes for patients with CF. She is regularly invited to media interviews and present at community, and as a keynote speaker at national and international meetings, on two occasions, and was the recipient of the best ECR research award at the 2019-Australian Academy of Science, Precision Medicine Conference. In 2020, the latter (co-culture airway organoid with pathogens) was pivoted to COVID-19 to delineate the innate immune response to SARS-CoV2 infection
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A/Prof Wendy Gold
The University of Sydney, Kids Research and The Children's Medical Research Institute

Case study: Rett syndrome – from diagnosis to treatment

2:30 PM - 2:45 PM

Biography

Associate Professor Wendy Gold is head of the Molecular Neurobiology Research group at Kids Research, The Children’s Hospital at Westmead, the University of Sydney. Her training includes an honors degree in Genetics and a PhD in Genetics and Immunology. Wendy conducts a research program of interdisciplinary translational research where she has contributed to the understanding of the underlying pathophysiology of many neurodevelopmental disorders as well as examining potential novel therapies. By utilising advanced techniques such as transgenic mouse models, brain organoids, and bioinformatics, her team is at the forefront of understanding and addressing these complex conditions. Her work aims to translate these innovative approaches into finding biomarkers and developing effective therapies such as gene therapies, offering hope for those affected by these challenging disorders.
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A/Prof Grant Logan
Children’s Medical Research Institute

Overcoming the challenges posed by pre-existing immunity to AAV viral vectors

2:45 PM - 3:00 PM

Biography

Associate Professor Grant Logan is a scientist at the Children’s Medical Research Institute with a research interest in translating gene therapies using vectors derived from adeno-associated virus (AAV). His research seeks to understand host-vector interactions and how this prevents long-term therapy as well as to leverage immunity as a powerful 'pressure point' to induce therapeutic responses. Grant has worked mainly in academia, but also in industry overseas. In addition to research projects currently underway, Grant holds an Honorary Hospital Scientist position in the Children’s Hospital at Westmead to build capacity for AAV serological services with the goal to providing a domestic service to screen for AAV gene therapy eligibility. Grant also holds a Federal Government appointment as a member of the Gene Therapy Technical Advisory Committee to the Office of the Gene Technology Regulator.
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A/Prof Wendy Gold
The University of Sydney, Kids Research and The Children's Medical Research Institute

Facilitator - Panel discussion: The Family Journey: Challenges and Opportunities in Rare Genetic Disease Care

3:00 PM - 3:30 PM

Biography

Associate Professor Wendy Gold is head of the Molecular Neurobiology Research group at Kids Research, The Children’s Hospital at Westmead, the University of Sydney. Her training includes an honors degree in Genetics and a PhD in Genetics and Immunology. Wendy conducts a research program of interdisciplinary translational research where she has contributed to the understanding of the underlying pathophysiology of many neurodevelopmental disorders as well as examining potential novel therapies. By utilising advanced techniques such as transgenic mouse models, brain organoids, and bioinformatics, her team is at the forefront of understanding and addressing these complex conditions. Her work aims to translate these innovative approaches into finding biomarkers and developing effective therapies such as gene therapies, offering hope for those affected by these challenging disorders.
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Mr Brett Anslow
Rett Research Australia

Panel member

Biography

Brett is a financial professional and father of three children. In 2016, his youngest daughter Holly was diagnosed with Rett Syndrome, a genetic rare disease, when she was three. Rett syndrome is a neurological disorder found almost exclusively in females. It effects around 1 in 10,000 births and there is no cure, yet. Since Holly’s diagnosis, Brett founded Rett Research Australia working with A/Professor Wendy Gold and her team, to raise funds to increase awareness and support of Rhett Syndrome medical research in Australia and around the world.
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Miss Lauren Jensen
Parent Advocate

Panel member

Biography

Lauren Jensen is a sustainability consultant, rare disease advocate, and mother to three-year-old Austin, who has STXBP1 disorder. Following Austin's diagnosis, she founded the STXBP1 Australia Foundation, where she serves as a board member and the Australian ambassador to the U.S. Foundation. Her efforts are focused on connecting families affected by STXBP1 to build crucial census data, now identifying 35 members nationwide. She provides this community with vital information and support in navigating the NDIS and healthcare systems. Additionally, Lauren is a member of Rare Voices Australia, where she advocates for increased access to clinical trials and early genetic testing for all individuals, regardless of their location or financial means. Through her efforts, she aims to foster a more inclusive and supportive environment for those impacted by rare genetic conditions, turning challenges into opportunities for better care and understanding.
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Ms Kris Pierce
Director Of Consumer Engagement
Child Unlimited

Panel member

Biography

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.
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