Children’s Medical Research Institute (CMRI) establishing Australia’s first clinical trial-scale facility

Children’s Medical Research Institute (CMRI) is Australia’s first dedicated paediatric medical research facility and has been helping to save the lives of children for over 60 years.

Right now, scientists at CMRI are helping to cure previously incurable genetic diseases through gene therapy research. Gene therapy fixes specific errors in DNA by applying knowledge of the human genome to clinical therapies. There are over 6000 different genetic diseases, and they affect 1 in 20 people. Ultimately, with a single injection gene therapy will scan the DNA, find an error, and correct it.

To conduct gene therapy research in Australia, researchers need timely access to vectors (biological tools for delivering genetic information). Currently, there is a waiting list of two years to obtain clinical-grade vectors from overseas manufacturing facilities, and this wait time greatly slows down the capacity of Australian research.

CMRI is working to establish Australia’s first clinical trial-scale facility to manufacture clinical-grade vectors for cell and gene therapy applications. This will create the ability to produce, in Australia, specific types of vectors that are suitable for use in human clinical trials for children and ultimately speed up clinical trials which can deliver improved health outcomes for Australian children.

Two internationally-renowned gene therapy researchers are leading CMRI’s gene therapy research: Professor Ian Alexander, who led Australia’s first gene therapy clinical trial for an inherited disease, and Dr Leszek Lisowski, who developed the world’s first bioengineered vector currently used gene therapy clinical trials. Dr Leszek is acutely aware of the importance of improving Australia’s vector manufacturing capabilities.

“The biggest bottleneck that slows down the translation of gene therapy tools to the patient is a global lack of vector manufacturing capacity, which significantly extends the timeline and increases the cost of translational studies,” he explained.

Families affected by genetic diseases are of course, desperate to speed up gene therapy clinical trials. When Julie Gravina found out her newborn twins, Isaac and Charlize, had a severe metabolic disorder called Propionic Acidemia, she generously donated liver cells to CMRI for gene therapy research.

“We’ve given Charlize’s liver cells to CMRI, and Professor Ian Alexander and his team are working on gene therapy for Propionic Acidemia right now. I think that is just so exciting, given the diagnosis that our children were given, and the outcomes. It’s more than you could ever wish for,’’ she said.

Find out more: https://www.cmri.org.au/Research/Research-Units/Gene-Therapy

This organisation is supported by trusts managed by Equity Trustees.