Exciting news today about the world-first delivery of a gene therapy at Westmead.
Researchers have for the first time calculated the lifetime cost of living with an inherited eye disease in Australia as $5.2 million per person. Distressingly, a significant portion of the cost associated with the progressive loss of vision is absorbed by the individuals affected and their families, with over one-third of these costs being dueContinue reading “Cost of going blind calculated for first time as $5.2m”
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”
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”
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”
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”
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”
CMRI has entered into a research collaboration with Zurich-based DiNAQOR, a genetic medicine platform company, to develop capsid gene therapy tools for treatment of both cardiovascular and kidney diseases.
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”
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”