National Gene Therapy Meeting
Recent findings for the progression of mRx-7 were presented at the Annual Joint Scientific Meeting hosted by the Australian Society for Stem Cell Research (ASSCR), Australasian Gene and Cell Therapy Society (AGCTS), and the International Society for Cell & Gene Therapy (ISCT) in Brisbane, Queensland, Australia. This conference, which spanned November 13 to November 15 2019, featured key speakers from around the world from broad fields of research including developmental biology, immunology, cancer, motor neuron disease and other rare disorders. This Conference highlighted how stem cells and gene therapies can be used to understand development, create disease models and develop new treatments. These areas dovetail with new advances in cellular therapies and regenerative medicine, and how these technologies are moving into the clinic. Complementing the idea of cell replacement, the conference will address advances in gene therapies, spanning work from vector development to clinical trials.
RNA therapeutics for cancer: are we there yet? – Presented by Prof Peter Leedman
Non-coding RNAs, including small interfering RNAs (siRNAs), microRNAs (miRNAs) and long non-coding RNAs (lncRNAs), are gaining prominence in clinical medicine as potential diagnostics and therapeutics. With the introduction of 2nd generation synthetic chemistry, the stability of short double stranded RNAs (dsRNAs, siRNAs and miRNAs) has been dramatically enhanced, producing profound effects on target gene expression, and leading to the FDA approval in late 2018 of the first siRNA drug, Patisiran. This landmark decision paves the way for subsequent dsRNA drug development. We have applied 2nd generation synthetic chemistry to develop a novel miRNA mimic for the treatment of epithelial cancers. In addition, we have engineered a liver-specific version of the miRNA mimic to treat hepatocellular carcinoma (HCC). The application of these emerging technologies to the treatment of cancer has opened up a range of new opportunities.