Leading the world in gene therapy research for spinal muscular atrophy
A world-leading research program has seen the Sydney Children’s Hospital, Randwick, be the first hospital globally to deliver a cutting-edge gene therapy for children up to six years old with spinal muscular atrophy (SMA).
Thanks to the outstanding work by the Kids Advanced Therapeutics (KAT) program, the hospital was successful in being chosen as a study site for the international clinical trial, dubbed the SMART trial.
The trial, dubbed the SMART trial, investigated the safety and efficacy of a gene therapy in a cohort of 24 young children...
Kids Research established its Research Quality Department to ensure that every research study involving children is safe, high quality, and family centred by streamlining governance processes, improving compliance, and strengthening patient and family engagement in research design and feedback.
They introduced a new research governance framework and clinical trial sponsorship process to speed up study start‑up and meet national quality standards. An internal Good Clinical Practice training program has built capability across the workforce, with more than 120 staff now trained to support high‑quality and consistent research.
The department also developed digital tools, including a Research Quality Management System and a Research Quality and Safety Dashboard, which track compliance, safety reporting, and consumer feedback in real time.
Together, these improvements have made research across the Network safer, more transparent, and more responsive to the needs of children and families.
Enabling sustainable clinical trial processes
Since establishing centralised clinical trial operations and business support, Kids Research has significantly strengthened the way we manage and support clinical research across our Network. The statewide‑mandated Clinical Trial Management System (CTMS), implemented April 2024, has provided the electronic infrastructure needed to reduce administrative burden, increase visibility of trial activity, and enhance financial oversight for the clinical trial portfolio. To date, we have engaged close to 30 clinical trial units, with authorised trials from early 2024 now fully built into and managed within the system.
The CTMS has enabled consistent workflows for study start‑up, financial management, and portfolio development, ensuring trial units can better capture revenue, clinical trial activity and streamline operations. Our CTMS Data Lead has played a critical role in maintaining data quality, supporting audits and reporting, and strengthening overall governance. Over the past year, Kids Research has activated more than 50 trials across 20 therapeutic areas, demonstrating sustained growth and improved research sustainability.
Transform paediatric organ transplantation
The Children’s Centre for Transplantation and Research (CCTR) is building the foundations for true bedside‑to‑bench‑to‑bedside research, establishing the partnerships and infrastructure needed to bring life‑changing discoveries directly into clinical care. Kindly supported by the Sydney Children's Hospitals Foundation, the CCTR has in recent months established partnerships with NSW Organ and Tissue Donation Service and LifeBlood, who will provide donated organs and blood products for research. Alongside our research partners, Children’s Medical Research Institute (CMRI), Westmead Institute for Medical Research (WIMR) and Western Sydney Local Health District, these collaborations are accelerating the translation of science into real-world outcomes for children and families.
In 2025, CCTR achieved a major milestone with the successful operation of its first paediatric perfusion machine and the maintenance of its first organ. A large team of multidisciplinary staff, including clinicians, surgeons, scientists and engineers have now completed foundational training to support future research and clinical translation.
Design and planning are underway for the purpose‑built paediatric perfusion lab, while research continues in an interim facility to maintain momentum toward next‑generation transplant innovation.
