Impact

• We developed the Adeno-associated virus (AAV) Gene Therapy Education Program, a multidisciplinary education program that has been listed on the Agency for Clinical Innovation (ACI) Innovation Exchange. It is designed to increase the knowledge and confidence of healthcare professionals who may be involved in delivering AAV gene therapy and/or are caring for patients who receive AAV gene therapy.
• We enabled SCHN to be gene therapy treatment sites for Spinal Muscular Atrophy (SMA) and inherited retinal disease. We continue to conduct groundbreaking trials on other rare conditions such as Duchenne Muscular Dystrophy (DMD) and will support new therapies as they emerge from the translational pipeline.
• Our KAT webinar series is how way keep the research community updated with the most up to date research on advanced therapeutics.
• We built the Advanced therapies handbook with support from our partners at UNSW and Brain-aid. It is a comprehensive educational resource on advanced therapeutics aimed at families and patients.
• Our advanced therapeutics 'Science Meets Healthcare' joint symposia, held in 2023, was another way Kids Research and partner, the Children's Medical Research Institute, brought together world-leading experts in advanced therapeutics to foster innovation and collaboration.

Program Manager

• Email: Michael.Shum@health.nsw.gov.au

Project Officer

• Email: Michelle.Kwok@health.nsw.gov.au