What it is
Precision Therapy has the potential to transform the lives of children suffering from rare genetic diseases, cancer and neurodevelopment diseases by offering potential cures.
NSW is the national leader in paediatric disease research and coordinated clinical trials networks. We enhance these capabilities by bringing together a uniquely skilled workforce to create a pipeline platform that bridges the gap between disease biology and the development for new drug candidates for paediatric diseases.
The Precision Therapy Platform focuses on two streams:
- The THerapeutic INnovations for Kids (THINK) program focuses on drug discovery by identifying novel and re-purposed therapeutics for childhood cancers, genetic diseases and neurodevelopmental disorders.
- The Kids Advanced Therapeutics (KAT) clinical trials program centres upon the development and delivery of early phase and advanced therapeutics clinical trials, the assessment of regulatory and sponsor risk for investigator-initiated trials, and the development of assessable frameworks and procedures to facilitate translation of clinical trials into clinical care. The program will initiate transformative Phase I/II clinical program trials to target diseases including liver diseases, blinding eye diseases and neurodevelopmental disorders.
- Children’s Cancer Institute (CCI)
- Sydney Children’s Hospitals Network (SCHN)
- University of NSW
- University of Sydney