Gene Therapy: Miracle in our Midst
Free Public Lecture
Level 1 Lecture Theatre
198 Berkeley Street
See more events from
Dr Elizabeth Finkel has documented the hope that was associated with the Human Genome Revolution and the Human Embryonic Stem Cells Revolution. After more than 20 years since they were announced, they have not had a significant impact in the clinic.
As editor in chief of a science magazine for six years, Dr Finkel is not sure how she missed the gene therapy revolution. Yes, she and her colleagues did the odd news report on gene therapy; but, mostly, they were blinded by the deluge of CRISPR publications – a cheap, precise new technique of gene editing that was transforming the ability to genetically modify plants, insects, animals and maybe one day humans. As it turns out, this happened in China last November.
But it wasn’t just Dr Finkel who missed the gene therapy revolution. It seems most people she has spoken to about it – including many medical people – hadn’t noticed.
This isn’t a cup half full story. The most dramatic example has biblical dimensions. In 2017, the New England Journal of Medicine published the results of a gene therapy trial for children born with Spinal Muscular Atrophy (SMA). These kids normally develop paralysis and die by the age of two. Instead, most were sitting and rolling; some were walking and talking. Is this just a fringe thing? Big Pharma doesn’t think so. Novartis recently paid $US 8.7 billion to purchase AveXis, the start-up company behind the SMA trial.
In this talk, Dr Finkel will guide us through the gene therapy revolution and how it is set to disrupt the way medicine is delivered.