The Kids Advanced Therapeutics Program (KAT) is leading the way to improve access to Advanced Therapeutics for children in Australia.
Advanced Therapeutics is a new class of therapies that are transforming the way we manage rare disorders in children and is the next evolution in healthcare, these include but are not limited to:
- Gene Therapy (in vivo & ex vivo)
- Somatic Cell Therapy
- Tissue Engineering
- Bacteriophage Therapy
We are national leaders in the delivery of Advanced Therapeutics through clinical trials and approved therapies to Australia children. Our teams are based across Sydney Children's Hospitals Network, the largest network of hospitals and services for children in Australia.
The strength and power of the translational pipeline of Advanced Therapeutics has never been more evident - our gene therapy trial success has changed the way we are able to treat children with Spinal Muscular Atrophy (SMA), our work in the gene therapy space has also been awarded the top project and idea by the National Health and Medical Research Council (NHMRC).
Building on the extraordinary success of the national SMA trial, we are working with major industry partners, research institutes and the NSW government to develop our infrastructure, organisational frameworks and workforce in order to further expand on our capability to deliver these therapies to children nationally and internationally.
