Australia’s first investigational gene therapy trials for dry age-related macular degeneration under way
Australia’s first clinical trials of an investigational gene therapy to treat dry age-related macular degeneration (AMD) have begun at the Centre for Eye Research Australia (CERA).
CERA’s Principal Investigator of Retinal Gene Therapy Research and vitreoretinal surgeon Dr Tom Edwards performed the first surgeries to administer the investigational gene therapy to patients at the Royal Victorian Eye and Ear Hospital in Melbourne.
This investigational gene therapy, being developed by Gyroscope Therapeutics Limited, is being studied in two studies evaluating its safety and effectiveness for the treatment of geographic atrophy (GA) secondary to AMD.
Age-related macular degeneration causes a gradual and permanent loss of central vision that worsens over time.
“Having the dry form of AMD is a devastating diagnosis which robs people of their ability to read, drive or even see the faces of loved ones,’’ says Dr Edwards.
“There is currently no approved treatment for dry AMD. As dry AMD advances, it leads to an irreversible degeneration of retinal cells, causing a gradual and permanent loss of central vision.
“We look forward to advancing important research for people facing devastating vision loss.”
Around 20 Australians with dry AMD are expected to take part in the HORIZON and EXPLORE trials, which are Phase II, multicentre, randomised, controlled trials evaluating the safety and effectiveness of an investigational gene therapy.
AMD occurs when debris develops in the central retina (or macula), the tissue responsible for taking light and turning it into vision.
In time the cells in the retina slowly die leaving gaps, or holes in the vision.
About one in seven Australians over 50 has the early signs of AMD (i), which can cause severe vision loss if it progresses to the late stage of the disease.
AMD is the leading cause of legal blindness in Australians over 50 (ii). There are two forms of late stage disease – wet and dry. Wet is where blood vessels bleed in the back of the eye.
As dry AMD advances, it leads to GA, an irreversible degeneration of retinal cells, causing a gradual and permanent loss of central vision.
For more information on the HORIZON trial [NCT04566445] or the EXPLORE trial [NCT04437368] please visit Gyroscope Therapeutics or Clinicaltrials.gov
Listen to Vision Australia Radio’s Peter Greco interview Dr Edwards on his recent work in AMD.
Janine Sim-Jones, Centre for Eye Research Australia, +61 420 886 511 firstname.lastname@example.org
(i) Mitchell, P., Smith, W., Attebo, K., & Wang, J. J. (1995). Prevalence of age-related maculopathy in Australia. The Blue Mountains Eye Study. Ophthalmology, 102(10), 1450-1460. Retrieved from
(ii) Foreman, J., Xie, J., Keel, S., van Wijngaarden, P., Sandhu, S. S., Ang, G. S., Dirani, M. (2017). The Prevalence and Causes of Vision Loss in Indigenous and Non-Indigenous Australians: The National Eye Health Survey. Ophthalmology, 124(12), 1743-1752. doi:10.1016/j.ophtha.2017.06.001