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Enabling Platform – Precision Therapy

Project Overview

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:

  1. 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.
  2. 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. 

Collaborations

  • Children’s Cancer Institute (CCI)
  • Sydney Children’s Hospitals Network (SCHN)
  • University of NSW
  • University of Sydney
Summary

The Precision Therapy enabling platform streamlines the path from scientific discovery to drug development, ultimately benefiting patients.

Lead Investigators

  • Dr Ian Street

    Professor Ian Street

    Director, THerapeutic INnovations for Kids (THINK), Children’s Cancer Institute (CCI), UNSW 
  • m lorentzos

    Dr Michelle Lorentzos

    Clinical Trials Medical Lead, Children’s Hospital Westmead, Sydney Children’s Hospitals Network
  • lani attwood

    Ms Lani Attwood

    Associate Director Research Operations, Kids Research, Sydney Children’s Hospitals Network

Project Research Team

THINK Investigators

Associate Professor Greg Arndt, Head of Drug Discovery, THINK; Head, Drug Discovery Centre,CCI

Associate Professor Mark Cowley, Deputy Director Enabling Platforms and Collaboration, CCI and the Project Lead for Advanced Genome and Transcriptome Analytics

Associate Professor Antoine de Weck, Group Leader, Computational Drug Discovery Biology, CCI

Associate Professor Paul Ekert, Deputy Director Research Themes, CCI

Ms Vanessa Tyrrell, Program Leader, Zero Childhood Cancer, Co-theme Head Personalised Medicine, CCI

Associate Professor David Ziegler, Clinical Trials Unit Head and Staff Specialist, Kids Cancer Centre at Sydney Children’s Hospital, Group Leader, Brain Tumours, CCI

Professor Robyn Jamieson, Head of Eye Genetics Research Unit at CMRI Head of Eye Genetics Clinics at The Children’s Hospital at Westmead and Westmead Adult Hospital

Professor Russell Dale,Head of Kids Neuroscience Centre, Children’s Hospital at Westmead,  Professor of Paediatric Neurology, University of Sydney

Dr Tim Failes, Senior Drug Discovery Officer, THINK  High Throughput Screening Facility Manager, Drug Discovery Centre, CCI

KAT Investigators

Adjunct Professor Paula Bray, Director Research, Sydney Children’s Hospitals Network

Dr Laura Fawcett, SCH Clinical Trials Lead, Sydney Children’s Hospitals Network

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Acknowledgment of Country.

In the spirit of reconciliation, the Luminesce Alliance acknowledges the Traditional Custodians of country throughout Australia and their connections to land, sea and community.

We pay our respect to their elders past and present and extend that respect to all Aboriginal and Torres Strait Islander peoples today.