Gene therapy trial success: game changer for children with SMA

New research has shown that gene therapy may provide an effective treatment for Spinal Muscular Atrophy (SMA), a devastating and fatal genetic condition. SMA affects the motor nerve cells in the spinal cord, causing progressive muscle weakness and preventing babies from being able to roll, sit up, crawl, walk and eventually breathe. Until recently, it was the leading genetic cause of infant death in Australia, occurring in 1 in every 10,000 births.

CMRI announces new collaboration with Gyroscope Therapeutics

Children’s Medical Research Institute and Gyroscope Therapeutics Holdings, a clinical-stage gene therapy company focused on treating diseases of the eye, today announced they have entered a research collaboration to develop next-generation clinical capsids, the protein shells of viral vectors used to deliver gene therapies. A team of researchers from CMRI and Gyroscope will work together in theContinue reading “CMRI announces new collaboration with Gyroscope Therapeutics”

Gene therapy project ranked top idea in Australia

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, in what the lead researcher describes as ‘proof that we weren’t just dreamers and symbolic of the power of the genomic revolution’. Professor Ian Alexander and his team were awardedContinue reading “Gene therapy project ranked top idea in Australia”

Announcing the establishment of the Australian Genome Therapeutics Centre

This collaborative effort 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.

Australia’s first gene therapy for genetic eye disease

The Therapeutic Goods Administration (TGA) has registered a drug known as LUXTURNA® for the first gene therapy for inherited retinal diseases in Australia. One of our senior scientists, Professor Robyn Jamieson, has been involved in the process to get to this exciting stage.