The extraordinary collaboration between clinical and discovery research that is moving new therapies into the mainstream was the theme of a Science Meets Healthcare joint symposia organised by Sydney Children’s Hospital Network (SCHN) and Children’s Medical Research Institute (CMRI) last month.

The two-day event featured speakers from across Australia as well as a special guest from the US, Professor Jeff Chamberlain, who is president of the American Society of Gene and Cell Therapy.

CMRI was represented on the first day by Professor Robyn Jamieson who is Head of the Eye Genetics Research Unit and is also Head of the Western Sydney Genetics Program at The Children’s Hospital at Westmead. Prof Jamieson spoke about the success of recent gene therapy in the eye.

“I think this is a very exciting space with lots of opportunities,’’ Prof Jamieson said. “Overall, it’s the teamwork that makes it happen.’’

On the second day, several CMRI scientists spoke including Associate Professor Samantha Ginn, Associate Professor Grant Logan, and Associate Professor Anai Gonzalez Cordero. They discussed gene therapy research into conditions ranging from OTC-deficiency, Spinal Muscular Atrophy and Usher Syndrome.

Associate Professor Leszek Lisowski spoke about his aim of finding treatments for children with rarer conditions which are often not part of bigger clinical trials.

“Imagine finding out your child has a cure available then finding out you can’t afford it,’’ he said. “We are trying to find solutions for rare disorders.''

Professor Ian Alexander, who is Head of the Gene Therapy Research Unit, reflected on his 30-year career and the excitement of what he has now seen come into the clinic.

“I do think that we are in a unique moment in the history of medicine that will change the face of medicine,’’ Prof Alexander said.

A key contributor to the research advances presented at the event was Luminesce Alliance, who , supported by NSW Health, fund and coordinate paeadatric precision medicine research between the Sydney Children’s Hospitals Network (SCHN), the Children’s Cancer Institute (CCI), the Children’s Medical Research Institute (CMRI), the University of NSW (UNSW), and the University of Sydney (USyd)..

Luminesce Alliance Executive Director, Anastasia Ioannou, said the event showed that lifechanging improvements can be achieved for children with rare genetic conditions when researchers and clinicians are working across multiple organisations and disciplines in the highly collaborative precision medicine environment.

“The valuable insights shared demonstrated the value of a multidisciplinary, collaborative approach to paediatric precision medicine,” Ms. Ioannou said.

The Science meets Healthcare joint symposia was organised by the Kids Advanced Therapeutics program at SCHN and CMRI, and was kindly supported by Westmead Institute for Medical Research, Luminesce Alliance, SCHF and NSW Health.