Gene therapy is presented on the global stage
World-leading gene therapy experts at Sydney Children’s Hospitals Network (SCHN) have shared their knowledge and experience on a global stage.
Researchers, Professor Ian Alexander and Associate Professor Michelle Farrar have spoken at separate conventions in California, after being honoured with an invitation to the events.
The pair led the only Australian clinical trial of gene therapy medication Zolgensma®, a breakthrough treatment for Spinal Muscular Atrophy (SMA), achieving life-changing results for babies diagnosed with the lethal condition.
Prof Alexander, Head of the Gene Therapy Research Unit, a joint initiative of The Children’s Hospital at Westmead (CHW) and Children’s Medical Research Institute (CMRI), attended the BIO International Convention in San Diego.
Regarded as the largest and most influential global event for the biotechnology industry, the Convention attracts thousands of biotechnology and pharma leaders, with an aim to discover new opportunities and promising partnerships, through a week of intensive networking.
Prof Alexander spoke at a session and associated panel discussion which showcased NSW’s leadership in Advanced Therapeutics and opportunities for industry engagement along the translational pathway, bringing promising new treatments from idea through to therapeutic reality.
“The session went very well with good attendance from industry delegates with strong positive feedback,’’ he said. “We also had many Australians in the audience who showed strong interest in increasing engagement with the NSW advanced therapeutics ecosystem.”
A/Prof Farrar, Paediatric Neurologist at Sydney Children’s Hospital, Randwick, was invited as an international speaker at the Cure SMA conference in Los Angeles, where she addressed a special session on how clinical observations inform future scientific questions and research.
“It was an amazing gathering of experts, who are looking at how to continue to improve advanced therapies and also towards the future, and don’t want to stop,” she said.
The annual Cure SMA conference brings together the world’s leading SMA researchers, clinicians and families living with SMA to network, learn and collaborate. In its 34th year, it’s the largest global conference for the SMA community.
The conference organises a variety of workshops and sessions, where researchers can reveal their latest advancements and the families and carers can build on a system of support and strength.