Kids Advanced Therapeutics

Kids Advanced Therapeutics

The Kids Advanced Therapeutics (KAT) program was born out of this multidisciplinary field aiming to support research, clinical application and the education of such new therapies. Advanced therapeutics includes gene therapy, somatic cell therapy, tissue engineered product, phage therapy and RNA therapies.

Advanced therapeutics is changing the landscape on rare diseases. These conditions had no treatment options previously and now the potential to treat and improve the quality of life of these children and their families is at our doorstep. This program envisions to provide access and continue to be the leader in the provision of these therapies.

The KAT program is proudly supported by Sydney Children’s Hospitals Foundation and Luminesce Alliance.


  • The KAT program has enabled the 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.
  • The KAT webinar series is a way to keep the research community updated with the most up to date research on advanced therapeutics. See more on our webinar page or SCHN YouTube channel.
  • With support from our partners at UNSW and Brain-aid, have built a comprehensive educational resource on advanced therapeutics aimed at families and patients.
  • The advanced therapeutics 'Science Meets Healthcare' joint symposia is another way Kids Research and its partner, the Children's Medical Research Institute, are bringing together world-leading experts in advanced therapeutics to foster innovation and collaboration.
Last updated Monday 22nd April 2024