Australian Genome Therapeutics Centre

Children’s Medical Research Institute (CMRI) has partnered with Sydney Children’s Hospitals Network (SCHN) to establish the Australian Genome Therapeutics Centre. It aims to transform the treatment of children with serious diseases using gene therapies.

The centre will offer a complete ‘pipeline’ “from design, construction, and testing of gene therapies, through to their production in a small-scale manufacturing facility, and on to treatment of patients with these new therapeutics within SCHN.

Read more about this unique partnership and the research opportunities ahead for our alliance partners here.


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.

The 3 year project to save infants’ lives, aims to exploit immunity to the AAV vector that is stimulated in infants receiving gene therapy for Spinal Muscular Atrophy, and to use this to engineer the next generation of vectors.

Professor Ian Alexander, Head of the Gene Therapy Research Unit, a joint initiative of Children’s Medical Research Institute (CMRI) and The Sydney Children’s Hospitals Network (SCHN), and Professor in Paediatrics and Molecular Medicine at the University of Sydney and his team’s work, over more than 25 years, has made significant contributions to the field of gene therapy and is now leading major advances in treatments for life-threatening genetic diseases.

Find out more about the exciting, innovative and transformational achievements of our alliance partners here.