Our vision is to continue to drive the development and accelerate the delivery of world-leading gene and gene-modified cell therapies to improve the health of all Australians.


The immense therapeutic power of the genomic revolution is beginning to be realised with stunning early successes in the treatment of genetic and acquired conditions, including cancer. The Australian Genome Therapeutics Centre (AGTC) has been established to accelerate the full realisation of this therapeutic power and builds on a legacy of collaboration between Children’s Medical Research Institute (CMRI) and The Sydney Children’s Hospitals Network (SCHN) that began with the establishment of a Gene Therapy Research Unit in 1995. This research effort has grown into a world-leading capability that spans the full translational pathway from idea to therapeutic reality that encompasses the development of novel gene transfer technologies and therapeutic strategies through to the implementation of ground-breaking clinical trials. The Centre brings together over 150 highly committed researchers and health care professionals with broad multidisciplinary expertise from discovery science to clinical implementation.  


Our Mission

  • Close the gap between the diagnostic and therapeutic power of the genomic revolution
  • Continue to push the boundaries of genomic technologies to bring an increasing number of paediatric and adult conditions within therapeutic reach
  • Integrate all elements of the translational pathway required to convert ideas into therapeutic realities
  • Target diseases, rare or common, genetic or acquired, that can be prevented, treated or cured using genomic technologies that repair, replace or modulate gene function
  • Consistently stay at the forefront of cutting-edge technology
  • Bring world best clinical trials to the Australian community at the earliest possible time
  • Train the next generation of clinicians and scientists along all steps of the translational pathway
  • Strategically engage and partner with Australian and International academia and commercial biotechnology sectors

The Partners

Sydney Children’s Hospitals Network (SCHN)

In 2019/20 a total of 153,027 children were cared for across the Network’s Westmead and Randwick campuses, including:

  • More than 50,000 inpatient admissions
  • More than 88,000 Emergency Department presentations
  • Close to one million outpatient service visits

The formation of the Network in 2010 was a significant milestone for paediatric services in NSW. The Network’s leadership group has established a solid corporate and strategic platform to enhance clinical care, further groundbreaking research, invest in education and training of the health workforce and advocate for a healthy future for children in NSW and beyond.

Our team, more than 8,000 staff across the Network, is committed to providing world-class paediatric health care in a family-focused, healing environment. Sick children have access to the best facilities, with speciality care available for children with conditions such as cancer, heart problems, severe burns, HIV/AIDS and children needing organ, bone marrow or cord blood transplants.

Sydney Children’s Hospitals Network has adopted the following purpose, mission and values to guide us in providing the best care for sick children and their families.

  • Purpose: helping children and young people live their healthiest lives possible.
  • Mission: Working in partnership to improve the health and wellbeing of children through clinical care, research, education and advocacy
  • Values: Collaboration, Openness, Respect, Empowerment

The Network’s legal name is The Sydney Children’s Hospitals Network (Randwick and Westmead) (incorporating the Royal Alexandra Hospital for Children)

ABN 53 188 579 090

Learn more https://www.schn.health.nsw.gov.au/research


Children’s Medical Research Institute (CMRI)

CMRI is dedicated to advancing the treatment and prevention of childhood diseases, so every child has the opportunity for a healthy start to life.

CMRI pioneered microsurgery, immunisations against lethal childhood illnesses and care for premature babies, all of which have improved the lives of countless Australian children over the last 60 years. Today, CMRI is the site of world-leading research in areas such as cancer, neurobiology, embryology, genomics and gene therapy.

Children’s Medical Research Institute (CMRI) actively conducts fundamental medical and biological research. Our scientists ask the difficult questions. What causes cancer? How can we stop it? Why does development go wrong and how can we prevent this? How does the brain work? How can what we’ve learned be used to treat cancer, epilepsy, and genetic diseases?

Our task is to make the future better

CMRI scientists ask the difficult questions in order to gain the important answers. This knowledge enables us to light the spark of discovery and to create a healthier future for all children.

What we’ve accomplished in the last 20 years:

  • Embarked on a world-first project to transform cancer diagnosis and personalise treatment planning within 7 years, part of the ‘Moonshot’ program to end cancer
  • Found a single genetic defect can cause cleft lip and palate
  • Fate map of the early embryo to help us understand many developmental problems
  • Identification of the components of telomerase, which will be important for treating 85% of all cancers
  • Partnering with The Sydney Children’s Hospital Network on a cure for genetic liver disease, with clinical trials imminent
  • Discovering and developing a new class of drugs for treating epilepsy

What we plan to accomplish in the next 20 years:

  • Develop epilepsy treatments that will help children (and adults) around the world
  • Develop new treatments for kidney disease and diabetes
  • Provide more gene therapy cures for inherited diseases in children
  • New treatments for infectious diseases
  • Telomere research to help us understand predisposition to diseases
  • Develop treatments targeting Alternative Lengthening of Telomeres, which occurs in some aggressive cancers that are currently most difficult to treat
  • Match cancer patients to the best treatment available

ABN 47 002 684 737

Learn More https://www.cmrijeansforgenes.org.au/


Contact Us

Australian Genome Therapeutics Centre

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