News & Events

WATCH: Advanced Therapeutics Webinar 8: A health system team approach to ocular gene therapy and vision rescue

02 August 2022
Gene therapy

Webinar series

In the 8th episode of our Advanced Therapeutics Webinar series we revisited the topic of ocular gene therapy with our biggest panel of speakers this year. Since LUXTURNA’s success as a world-first gene replacement therapy for an RPE65-related retinal vision loss early last year, our researchers have made great strides in paving the way for the treatment of patients with all kinds of...

Funding boost for cutting-edge health treatments

24 June 2022
Gene therapy

Viral Vector Facility

Kids Research welcomes the NSW government’s funding boost for biomedical research, putting the state at the forefront of cutting-edge health treatments. The Perrottet government has secured $270 million, announced in the state budget, to go towards two new facilities; the Viral Vector manufacturing facility at Westmead and the Sydney Biomedical Accelerator Complex in Camperdown. Premier Domini...

SCHN gene therapy experts speak at global events

21 June 2022
Gene therapy

Gene therapy

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 th...

World-first treatment changes outlook for SMA babies

18 June 2022
Gene therapy

World-first SMA treatment

Babies diagnosed with Spinal Muscular Atrophy (SMA) at birth who received life-changing gene therapy are now celebrating their first birthday symptom-free or with minimal symptoms thanks to Sydney Children’s Hospitals Network’s (SCHN) world-first clinical trial. The SPR1NT trial, published in the prestigious journal Nature Medicine (two copies of SMN2 and three copies of SMN2), successfull...

The world's most expensive drug now free of charge

30 April 2022
Gene therapy

Gene therapy

At four months of age Sophia is reaching all her developmental milestones. She demonstrates good head control while sitting supported and can even roll from her tummy to her back. Her parents Kaitlin and Max have not witnessed any decline in Sophia’s physical development and for this they are extremely grateful to the Paediatric Neurology team at Sydney Children’s Hospital, Randwick (SCH)....

New facility in Western Sydney to deliver life-saving medical technology

31 January 2022
Gene therapy

Gene Therapy

Children with genetic diseases and cancers will be given faster access to life-saving gene and cell therapies through a new commercial scale viral vector manufacturing facility in Western Sydney. The NSW government has called on industry and investors to submit expressions of interest to support the growth and operations of the facility, to be built in the Westmead Health and Innovation Distri...

Researchers discover a new pathway to ocular gene therapy

20 December 2021
Gene therapy

Ocular gene therapy

Two young sisters with a blinding eye condition will now have access to ocular gene therapy after a new pathway to the treatment was discovered. The girls, who were diagnosed with early-onset severe retinal dystrophy (EOSRD), an inherited degenerative disease of the retina, began having trouble with their night vision at four and two years of age. With few effective treatments available for...

The establishment of Australian Genome Therapeutics Centre

16 August 2021
Gene therapy

Gene Therapy

The Sydney Children’s Hospitals Network (SCHN) and Children’s Medical Research Institute (CMRI) are pleased to announce the establishment of the Australian Genome Therapeutics Centre (AGTC), 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...

Gene therapy project ranked top idea in Australia

16 August 2021
Gene therapy

gene therapy

A gene therapy project to save infants’ lives has been named the top-ranked National Health and Medical Research Council (NHMRC) Ideas Grant for 2020. The project led by Professor Ian Alexander and his team were awarded the 2020 NHMRC Marshall and Warren Ideas Grant Award at the NHMRC Research Excellence Awards Dinner on June 16th. Professor Ian Alexander, Head of the Gene Therapy Research Uni...

Gene Therapy – The Answer to Childhood Genetic Diseases?

19 June 2019
Gene therapy

Gene Therapy

The need for effective treatments for children with rare genetic diseases has been a long and challenging road in research and the community. With the advancement of technology, research is now a step closer to an answer – gene therapy. A major game changer in medicine, gene therapy was recently addressed at the National Press Club of Australia by Dr Elizabeth Finkel, who is the first journali...

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