Strong clinical and research integration Enabling the rapid and safe translation of advanced therapeutics into patient care through integrated clinical, academic, and governance processes, which are underpinned by specialised infrastructure (e.g. PC2 laboratories supporting GMO preparation).
Collaborative and multidisciplinary culture Embedding collaboration between clinicians, researchers, governance experts, and operational teams. These teams are supported by GCP trained staff and the Gene Therapy Multi-disciplinary team to ensure the safe, consistent, and efficient delivery of advanced therapeutic clinical trials.
Established governance and regulatory leadership Demonstrating strong regulatory leadership and streamlining approval processes to accelerate clinical trial start‑up. Establishing an overarching OGTR licence has positioned SCHN as a globally competitive advanced therapeutics clinical trial site.
Reputation and network influence Leveraging national partnerships with government, regulators, academic, and industry stakeholders to consistently deliver paediatric advanced therapeutics, advocate for patients, and lead in policies and research.
Our top four capabilities
Clinical trials enablement and delivery Facilitating timely, patient‑centred delivery of advanced therapeutic clinical trials by streamlining regulatory pathways, coordinating early‑phase trial activation, and working closely with ethics, governance and clinical trials teams.
Workforce capability and education Building capability through targeted training and knowledge‑sharing that support the safe, ethical, and efficient delivery of advanced therapies, including biosafety training, comprehensive operating procedures, and tailored education for staff, patients, and families.
Multidisciplinary clinical care integration and gene therapy expertise Cultivating cross‑disciplinary collaboration of experts (i.e. Trial Enabling Flying Squad or TEFS) in the field to ensure new trials are reviewed rapidly, selected ethically, and conducted safely and effectively. Demonstrating the importance of highlighting the voice of people with lived experience in clinical trial co-design and informing outcome measurements.
Strategic partnerships and system readiness Strengthening healthcare system readiness through strategic partnerships supporting sustainable infrastructure, governance and translational pathways, including a delegated Institutional Biosafety Committee that manages GMO‑related risk and oversees PC2 compliance to interface with the OGTR.
Types of advanced therapeutic capabilities at Kids Research
AAV gene therapies
Stem cell therapies
CAR T-cell therapies
Tissue-engineered products
Phage therapies
RNA-based therapies
Antisense oligonucleotides
Other personalised precision therapies
Successes we celebrate as an organisation in our advanced therapeutic expertise
We led the activation of complex pharmaceutical-sponsored and investigator-initiated early-phase and advanced therapeutics trials across multiple specialties, including neurology, genetic metabolic disorders, ophthalmology and nephrology.
We became the first hospital globally to deliver a novel gene therapy for children up to six years of age with spinal muscular atrophy (SMA).
We successfully led the Newborn Screening Program pilot for spinal muscular atrophy (SMA) and primary immunodeficiencies across NSW and ACT, contributing to SMA being adopted into Australia’s national newborn screening program.
We secured an overarching OGTR DNIR licence for paediatric clinical trials utilising adeno-associated viral (AAV) vectors, significantly streamlining governance for gene therapy trials.
We developed Australia’s first multidisciplinary AAV Gene Therapy Education Program for healthcare professionals and complementary Advanced therapies handbook to support families considering or undergoing gene therapy.
We present a Kids Advanced Therapeutics educational webinar series that features world-class experts sharing their current and future work exploring how advanced therapies are shaping the future of paediatric health care.
We established and lead the national Australian Network of Paediatric Trial Centres (ANPTC), strengthening national partnerships, research translation, and clinical trial capability.
We are leading the development of a multidisciplinary framework for patient-centred delivery of advanced therapeutics in genetic disease.
We are collaborating with patients and families with lived experience to partner in research and advocacy.
We were a finalist in the 2025 NSW Health Awards (Health Research category), recognising leadership in paediatric advanced therapeutics.
