New centre set to transform treatment for sick kids

Sydney Children's Hospitals Network (SCHN) and Children's Medical Research Institute (CMRI) are announcing the establishment of the Australian Genome Therapeutics Centre (AGTC), a collaborative effort which will transform the treatment of children with serious inherited diseases and contribute to the development of exciting new treatment options for a wide range of other diseases, including cancer, across all age groups.

Genome therapeutics, often referred to as gene therapies, encompass a number of new types of treatment that use genes as medicines, correcting diseases at their source.

Establishment of this Centre represents a major step forward in a journey that began 26 years ago with a small number of clinicians and scientists in the joint gene therapy research program of SCHN and CMRI.

At the start of this journey, many scientists regarded the idea of gene therapy as science fiction. Now, several highly successful gene therapy trials have demonstrated the immense potential of this approach, and SCHN and CMRI have more than 150 researchers working in this revolutionary new area of medicine.

The researchers have made major contributions to the international effort to develop gene delivery systems that are essential for successful gene therapy. These include viral vectors (viruses that have their genetic material replaced by a genetic medicine) and lipid nanoparticles similar to those used to deliver mRNA in the Pfizer and Moderna COVID-19 vaccines.

“This unique partnership between clinicians and researchers gives us the ability to make a life-changing difference to the children and families who currently don’t have answers. It gives us the opportunity to offer world-leading clinical trials to paediatric patients across NSW as soon as possible and the potential to develop new treatments, and even cures, that can help children both now and in the future. It is truly a transformational approach to healthcare,” Chief Executive of SCHN, Ms Cathryn Cox, said.

Professor Ian Alexander is one of the leaders of the AGTC. He is Head of the Gene Therapy Research Unit, working across both CMRI and SCHN and is considered a world-leader in this field.

“Excellent genomic diagnostic work is being done all around Australia, and we’ve always punched above our weight internationally in this, but the main challenge has been translating that research into the clinic,’’ Professor Alexander said.

“Now, finally, we will be able to go beyond offering a diagnosis. It’s very exciting because now we’re working with an increasing number of diseases that are within reach of a treatment or cure.’’

The researchers in AGTC are developing and delivering treatments for conditions including inherited metabolic liver disease, inherited causes of blindness, lung diseases, brain diseases and serious neuromuscular disorders. They and their colleagues also have expertise in development and delivery of CAR T treatments, another form of gene therapy, for cancer.

Future applications of this technology will include treatment of many common conditions such as heart disease, and macular degeneration - a common cause of blindness in older people.

“This will be a collaborative effort with researchers elsewhere in NSW, other Australian States, and internationally. The researchers in AGTC are providing a complete 'pipeline' from design, construction, and testing of gene therapies, through to their production in a small-scale manufacturing facility, and on to treatment of patients with these new therapeutics within SCHN," CMRI's Director, Professor Roger Reddel, said.

"This effort will be advanced even further by the Viral Vector Manufacturing Facility to which the NSW Government has committed major funding."

Learn more on the Australian Genome Therapeutics Centre website.