Cure The Future is proud to have supported researchers at Sydney’s RPA as part of an international team to have developed a gene therapy for the life-threatening blood disorder, haemophilia. The breakthrough, led by Professor John Rasko, is a major milestone in the quest to cure the bleeding disorder and to the possibility of treating over 4000 other genetically inherited diseases.
The U.S. Food and Drug Administration today approved a 2nd cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. “Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases” said FDA Commissioner Scott Gottlieb, M.D.
Your Cure The Future donations have supported a ground-breaking discovery which may help find new treatments for cancer and other diseases. Dr Ulf Schmitz and Centenary Institute’s Gene & Stem Cell Therapy Program, led by Professor John Rasko AO, have – for the first time – discovered a common pattern in the process of gene regulation in humans, mice, dogs, chickens and zebrafish. A better understanding of this common process will help develop novel therapies and possible cures for diseases.
Car-T Therapy has been approved by the FDA in America – the first ever approval of a Cell-based Gene Therapy, which Cure The Future was set up to support. We are delighted that Cell & Gene Therapy is finally approved as a treatment in the USA and remain committed to fund further research to find a cure for the thousands of other diseases we can inherit through our DNA.