Prestigious national award for Professor Ian Alexander

A pioneer of the Australian gene therapy field, Professor Ian Alexander, has been awarded the prestigious Peter Wills Medal as part of the Research Australia Health and Medical Research Awards. Professor Alexander is head of Children’s Medical Research Institute’s Gene Therapy Research Unit, which he founded over 25 years ago, as a joint initiative withContinue reading “Prestigious national award for Professor Ian Alexander”

New funding puts Viral Vector Manufacturing Facility in winning position

Children’s Medical Research Institute warmly welcomes the NSW Government’s announcement of its allocation in the 2022/23 Budget of $101.4m toward a commercial-scale Viral Vector Manufacturing Facility. This significant investment will greatly advance the ability to treat children with serious genetic diseases. In addition to funds to build the manufacturing facility, the $101.4 million commitment includesContinue reading “New funding puts Viral Vector Manufacturing Facility in winning position”

Gene Therapy Improvement Has Massive Potential

A team of scientists from Children’s Medical Research Institute (CMRI) in Sydney have developed a new way to improve targeting of specific organs and tissue types in gene therapy – making this innovative gene delivery technology more efficient and described as having “massive potential’’ for the field. The work is featured as the cover storyContinue reading “Gene Therapy Improvement Has Massive Potential”

Partnership for RNA Project

Scientists from Children’s Medical Research Institute (CMRI) have partnered with RNA researchers from the Monash Institute of Pharmaceutical Sciences (MIPS) to combine mRNA delivery with viral gene delivery to treat metabolic liver disease in infants and children. The partnership will be funded by a $99k grant from the mRNA Victoria Research Acceleration Fund along with grantsContinue reading “Partnership for RNA Project”

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

CMRI is delighted by the NSW Government’s announcement about the establishment of Australia’s ​first commercial-scale viral vector manufacturing facility in the Westmead Health and Innovation District. The ability to manufacture high-quality (clinical grade) viral vectors in Australia is critically important for our gene therapy programs. It will accelerate the delivery of, and access to, life-saving new gene therapies developed in the CMRI labsContinue reading “New research facility to deliver life-saving medical technology in Western Sydney”

Australian first gene therapy for childhood blindness

Two Sydney siblings have become the first patients in the country to receive a novel gene therapy that has rescued their vision and holds hope for preventing them from going blind. The ocular gene therapy, LUXTURNA, is the world’s first approved gene replacement therapy for an inherited blinding eye condition and one of the firstContinue reading “Australian first gene therapy for childhood blindness”

CMRI Awarded Multiple Medical Research Future Fund Grants

Children’s Medical Research Institute (CMRI) was awarded multiple Medical Research Future Fund (MRFF) grants to help improve the lives of children living with genetic diseases. The MRFF, which is an initiative of the Australian Government, has funded research projects in cancer, gene therapy, and stem cell medicine at CMRI. Dr Anai Gonzalez-Cordero, head of theContinue reading “CMRI Awarded Multiple Medical Research Future Fund Grants”

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”