Gene Therapy
The Gene Therapy Research Unit finds ways to correct genetic diseases in children.
Research Overview
The Gene Therapy Research Unit is a joint initiative of Children’s Medical Research Institute and Kids Research based at The Children’s Hospital at Westmead (CHW). Our primary goals are to develop more effective gene therapy methods and to translate basic research progress into improved health outcomes for children.
We develop next-generation gene therapies designed for real clinical impact, from precision genome editing to advanced AAV delivery systems. Our work bridges discovery and translation to create safe, durable treatments for metabolic, neuromuscular, and endocrine disorders.
Realising the therapeutic potential of progress in the laboratory is our ‘raison d’etre’, so we are particularly pleased to have three clinical trials under development or close to initiation. This is a major achievement and places our group at the forefront of this exciting field in Australia.”
Professor Ian Alexander
Head of the Gene Therapy Research Unit
Team Members
Research Projects
Together, our research advances next‑generation gene therapies through innovations in AAV vector engineering, immune‑response profiling, targeted delivery, and precision genome editing.
Across our collective work, we are expanding therapeutic possibilities for metabolic, neuromuscular, and endocrine disorders by improving gene‑editing technologies, refining AAV capsid design, and developing novel delivery systems that enhance safety, specificity, and overall vector performance.
Our recent studies span preclinical genome‑editing rescue, immune‑barrier characterisation, and clinical genomic testing, positioning our group at the forefront of translating emerging genetic technologies into durable, clinically meaningful treatments.
Former Team Members
Grant Logan - A/Prof
Bradley Hall - PhD Student
Ann-Maree Huang - PhD Student
Imogen Astruc - PhD Student
Neeta Khandekar - Research Assistant
Kate Mullany - Post PhD Intern
Sophia Liao - Senior Research Assistant
Publications
PubMed
Prof Ian Alexander publication list
PubMed
A/Prof Samantha Ginn publication list
Major Achievements
1995
Gene Therapy Research Unit formed as a joint initiative of CMRI and The Children’s Hospital at Westmead.
2000
Established clinical grade gene transfer vector production capacity in Australia.
2002
In collaboration with the Children’s Hospital in Paris, conducted first ever gene therapy clinical trial for a genetic disease in Australia.
2009-11
In collaboration with the Australian pharmaceutical industry, prepared a gene transfer vector and anti-cancer drug formulation for paediatric clinical trial use.
2018
Successfully worked with NSW Health to have Spinal Muscular Atrophy (SMA) added to newborn screening.
2019
Began Australian trial for SMA gene replacement therapy.
2020
Contributed to national frameworks guiding safe and ethical clinical translation of gene and cell therapies in Australia.
2021
Advanced next generation AAV vector technologies with improved targeting and safety profiles.
Expanded clinical gene therapy programs for rare childhood genetic and metabolic diseases.
2022
Prof Ian Alexander was awarded the Peter Wills Medal for outstanding contribution to building Australia’s international reputation in the area of health and medical research and fostering collaboration for better health.
2024
Developed new preclinical models for liver directed gene therapy to support future clinical translation.
2025
Australia’s first clinical and commercial-scale viral vector Contract Development and Manufacturing Organisation (CDMO) was established by SCHN with Prof Alexander.