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Symposium 12: Novel inhalable gene and drug delivery strategies to combat respiratory diseases

Tracks
Track 4
Wednesday, December 4, 2024
10:30 AM - 12:30 PM
Courtyard Room 1&2

Details

The purpose of this symposium of to discuss a broad range of novel ‘localised’ pharmacological and mechanical solutions to problems surrounding the existing treatment of respiratory diseases with small molecule drugs (via oral or inhaled delivery). Solutions that are actively being explored worldwide at the moment include inhaled nanomedicines (that control drug exposure in the lungs and systemically), inhaled gene therapy and inhaled vaccination. This symposium will collectively discuss the utility of novel localised strategies that include inhaled ‘nanomedicine’ approaches (including bacteriophages and nanoparticles) and the impact of intentional and disease induced airway perturbations on the efficacy of inhaled gene therapy. Importantly, a large number of researchers in Australia and worldwide are currently examining inhaled gene delivery strategies to treat genetic diseases that affect the lungs, such as cystic fibrosis. The first speaker has therefore been selected to discuss and compare the efficacy and adverse effects of inhaled gene therapy versus small molecule protein modulators as treatments for CF based on her recent research. Respiratory diseases that will be discussed include genetic diseases (speakers 1 and 2) as well as bacterial (speaker 3) and viral (speaker 4) infections.


Speaker

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Dr Shafagh Waters
UNSW Sydney

Lentiviral gene therapy and stem cell transplantation in cystic fibrosis: A comparative analysis with CFTR modulator therapy

10:30 AM - 11:00 AM

Abstract document

Biography

Dr. Shafagh Waters (BSc, MSc (Disc.), PhD) is a Scientia Senior Lecturer at the University of New South Wales (UNSW) and an Honorary Senior Scientist at Sydney Children’s Hospital. She completed her PhD at the Australian National University (ANU), followed by postdoctoral fellowships at UNSW from 2013 to 2016. During this time, she received prestigious international fellowships for gene therapy research at City of Hope in the USA and training in organoid medicine in Lisbon, Portugal. She established her independent research lab in 2016. Dr. Waters leads an NHMRC-funded research program focused on adult stem cell biology for cystic fibrosis (CF), supported by 32 grants, 21 of which she serves as Chief Investigator (CIA), including partnerships with national and international industry collaborators. Her work has led to the development of an Australian national biobank for stem-cell-derived airway and gut organoids, a high-throughput platform for testing therapeutic interventions on patient-derived organoids, and the implementation of organoid-guided clinical trials for cystic fibrosis. In these trials, patient-derived organoids are used to identify the most effective treatments, directly guiding personalized therapy for CF patients. A founding member of the NSW Non-Animal Technologies Network, Dr. Waters combines her expertise in organoid disease modeling with advanced work in creating complex organoids that incorporate vascularization and immune cells. Her research also includes developing co-culture systems to study interactions between airway organoids and pathogens. During the COVID-19 pandemic, this approach was adapted to explore the innate immune response to SARS-CoV-2, providing crucial insights into host-pathogen interactions. In recognition of her contributions to science and public engagement, Dr. Waters was awarded the NSW Young Tall Poppy Science Award in 2022, acknowledging her outstanding research achievements and commitment to science communication.
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Dr Alexandra McCarron
Adelaide Uni and the Womens and Childrens Hospital, Adelaide

Impact of airway surface pertubations and infection on the efficacy of inhaled gene therapy

11:00 AM - 11:30 AM

Abstract document

Biography

Dr Alexandra McCarron is a research fellow in the Cystic Fibrosis Airway Research Group at the University of Adelaide. She is passionate about transforming the lives of those with cystic fibrosis and improving their quality of life and longevity. Dr McCarron’s research is focussed on advancing lung-targeted genetic therapies for cystic fibrosis. She is currently developing novel ways to enhance the delivery of these therapies, aiming to improve their overall effectiveness.
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Prof Hak-Kim Chan
The University of Sydney

Inhalable bacteriophage therapy to treat respiratory diseases

11:30 AM - 12:00 PM

Abstract document

Biography

Hak-Kim Chan, Professor in Pharmaceutics, is leading the Advanced Drug Delivery Group at the Sydney Pharmacy School, University of Sydney. Kim is a world leader in respiratory drug delivery, with a research program ranging from powder production by novel processes, particle engineering and aerosol formulation, to scintigraphic imaging of lung deposition and clinical outcome. He is a Fellow of American Association of Pharmaceutical Scientists, was Vice President of Asian Federation for Pharmaceutical Science, Executive Editor of Advanced Drug Delivery Reviews, and recipient of the 2023 Career Achievement Award of The International Society for Aerosols in Medicine.
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Prof Clive Prestidge
Director Centre For Pharmaceutical Innovation
University of South Australia

Next Generation Carrier Particles for Therapeutics Delivery to the Lung: Challenges and Opportunities

12:00 PM - 12:30 PM

Abstract document

Biography

Clive Prestidge is the University of South Australia’s Professor of Pharmaceutical Science, leading the internationally recognised Nanostructure and Drug Delivery research group and co-Director for the Centre for Pharmaceutical Innovation. He has an extensive track record in nanomaterials for drug delivery and the development of nano-biomaterials for outcomes in Health and Medicine. He has supervised over 50 PhD and MSc students and authored around 300 international journal articles. He is an inventor of several technology platforms, and has been extensively involved in patenting, licencing, spin off companies and partnering with industry to advance commercialisation/clinical translation.

Chair

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Shadab Haque
Monash University-MMIC

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Lisa Kaminskas
The University of Queensland

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