Advanced therapeutics is rapidly emerging as a field with mass potential in the science and healthcare landscape, opening up new possibilities for childhood diseases that were once considered incurable. Helping to lead the charge in this space are the teams at Sydney Children's Hospitals Network and the Children's Medical Research Institute.

Recently, the powerhouse minds driving this innovation came together in the second annual joint symposia, showcasing the breakthroughs that have already been achieved through advanced therapeutics and where the future of medicine is headed.

The two-day event, appropriately themed 'Science meets Healthcare', hosted discussions by leading national and international experts and their clinical counterparts on the latest scientific developments in paediatric advanced therapeutics, including the impact of gene therapy on rare diseases like Spinal Muscular Atrophy (SMA), Inherited Retinal Blindness and Duchenne Muscular Dystrophy (DMD) and the science behind these novel treatments.

The lineup included Professor Jeff Chamberlain, President of the American Society of Gene and Cell Therapy, who presented on the current science of gene therapies for DMD, and outlined the 20-year history behind gene therapy and the basic science of new gene therapy delivery methods.

Through the Kids Advanced Therapeutics program, SCHN has already become a national leader in the delivery of advanced therapeutics, and has earned a reputation internationally as a site that can participate in groundbreaking clinical trials and deliver novel approved therapies for children with rare diseases.

“Five years ago, through the initiative of Professor Michelle Farrar, we treated the first Australian baby using gene therapy. Since then, we have become an accredited treatment centre for two rare conditions—spinal muscular atrophy and inherited retinal blindness,” Cathryn Cox, Chief Executive of SCHN, said in her opening remarks.

Professor Michelle Farrar, a paediatric neurologist at Sydney Children's Hospital, Randwick (SCH), said advanced therapeutics are no longer science fiction and iterated the importance of being ready for the next groundbreaking development.

“Research is not a linear process. From basic science to clinical research to clinical practice, we need to be ready,” Prof. Farrar said.

Professor Robyn Jamieson, Head of the Western Sydney Genetics Program at The Children’s Hospital at Westmead (CHW) and Head of the Eye Genetics Research Unit at CMRI, added that while research and science are helping to pave the way, it is collaboration that makes each new success possible.

“We are in the era of genetic therapies and this is a very exciting space. But I think overall it’s the teamwork that makes it happen,” Prof. Jamieson said.

The symposia was proudly supported by Sydney Children’s Hospitals Foundation (SCHF) and Luminesce Alliance, which have both had long-lasting relationships supporting clinical trials of advanced therapeutics and their translation into clinical care at SCHN, along with the Westmead Institute for Medical Research and NSW Health.

“It can take up to 17 years to translate research to clinical care–that’s an entire childhood. By working hand in hand, we can fast track transformative care from the bench to the bedside and make a bigger impact on children’s health sooner,” Kristina Keneally, Chief Executive Officer of SCHF said.

Anastasia Ioannou, Executive Director of Luminesce Alliance, commented how events like these highlight the value of partnership.

“The valuable insights shared demonstrate 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.