New view of gene therapy
CERA’s newest start-up company is taking a novel approach to gene therapy, aiming to ‘switch on sight’ by restoring light-sensing cells.
More than 190 million people worldwide have retinal diseases where death of photoreceptors – cells at the back of the eye that sense light – leads to vision loss and blindness.
Currently there is no way to restore lost vision after photoreceptors die, but CERA’s newest start-up company Mirugen is aiming to regenerate the lost cells to preserve and restore visual function.
Mirugen comes from the Japanese word ‘Miru’ for ‘view’ and gen for ‘gene therapy’.
It acknowledges the Japanese origins of Nobel Prize-winning cellular reprogramming research – which has now been adopted by research teams around the world.
“Cellular reprogramming is a technology which allows you to control genes to determine how cells behave,” Associate Professor Wong says.
The process uses engineered genes to turn Müller glia cells in the retina into new photoreceptor cells to replace those lost in disease.
In chicken and fish, Müller glia cells can mobilise and repair the retina.
“This ability has been lost or suppressed in mammals including humans,” Associate Professor Wong says.
“We’re working to unlock this regenerative ability.
“Many diseases with damaged photoreceptors could potentially benefit from this, including age-related macular degeneration, retinitis pigmentosa and Stargardt’s disease.”
Mirugen is the third start-up company to be incubated at CERA – following ophthalmic referral platform Oculo (2015) and Enlighten Imaging (2019), which is developing a simple eye scan to detect the early signs of Alzheimer’s disease and a variety of other conditions.
Mirugen is also one of the first biotechnology start-ups to receive seed funding from Australia’s biotech incubator CUREator, which is backed by the Australian Federal Government’s Medical Research Future Fund.
Mirugen Director and CERA Managing Director Professor Keith Martin says developing start-up companies is an important part of CERA’s strategy.
“The unique environment which sees lab-based research occur alongside clinical research provides a direct runway for taking promising pre-clinical research like Mirugen’s through to clinical trial,” says Professor Martin.
This article first appeared in See the future: Annual Review 2022.