Professor Elizabeth Rakoczy is modifying viruses to use their powers for good. She’s created a new gene therapy for wet age-related macular degeneration (AMD) that is reversing vision loss in clinical trial patients. Her treatment means one injection instead of several per year.
Modified viruses are gene therapy’s delivery vehicles, taking genes directly into cells. Elizabeth first showed that they could carry a healthy replacement for a mutated gene that causes degeneration of the eye’s retina. She then showed they can deliver instructions for eye cells to form a bio-factory to produce their own treatment for wet AMD, a more complex eye disease.
More than 112,000 Australians have wet AMD—the most devastating form of AMD—and up to 8,000 more commence treatment for it each year. Each injection of the current treatment costs about $2,000, and patients have six to eight per year. Costs will rise with Australia’s ageing population. Gene therapy offers an alternative treatment.
Elizabeth hopes to adapt her bio-factory idea to other diseases to alleviate suffering.
Elizabeth’s gene therapy replaces the monthly injections with a single one. Once established within the eye cells, the bio-factories start producing the medication, a naturally occurring molecule that mops up the rogue VEGF. With VEGF removed, the blood vessels diminish and the macula recovers. And because the bio-factories remain operational, potentially for years, the patient’s eyesight is maintained.
This therapy has been licensed to US company Avalanche Biotechnologies Inc., which has raised over $400 million to progress the treatment through clinical trials and bring it to market. This makes Elizabeth one of the few Australians to successfully take a research idea from ‘bench to bed’ and commercialise it internationally.
Watch a video about her work here