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Gene therapy trial for dry AMD under way in Australia

Australia’s first clinical trials of an investigational gene therapy for dry age-related macular degeneration (AMD) has commenced 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 Gyroscope Therapeutics’ gene therapy, GT005, to patients at The Royal Victorian Eye and Ear Hospital in Melbourne recently.

The therapy – delivered with a subretinal injection – is featuring in two studies called HORIZON and EXPLORE to evaluate its safety and effectiveness in treating geographic atrophy (GA) secondary to AMD.

Around 20 Australians with dry AMD are expected to take part in both trials, which are Phase 2, multicentre, randomised, controlled trials.

According to the company, GT005 is designed to stimulate a person’s cells to create Complement Factor I (CFI), a protein that is key to regulating the alternative pathway of the complement system. The goal is to slow the progression of dry AMD.

“To deliver the gene therapy, the gene is combined with a safe, modified virus – known as a viral vector – which help it get into retinal cells,” Edwards said.

“The therapy aims to increase the production of [CFI] which regulates part of the immune system, known as the complement system. Too much activity in the complement system, has been strongly associated with the development of AMD.

“And it’s believed that increasing the production of this protein could dampen the system’s overactivity and reduce inflammation, with the goal of preserving a person’s eyesight.”

Gyroscope Therapeutics’ EXPLORE trial is enrolling up to 75 people globally with GA secondary to AMD who have rare variants in their Complement Factor I (CFI) gene associated with low levels of the CFI protein in their blood.

HORIZON is evaluating GT005 in a broader group of up to 180 people with GA secondary to AMD.

Participants receive two doses of GT005 administered as a single subretinal injection. Participants may be assigned to one of two treatment arms or the control arm of the trial and will be followed for 48 weeks.

EXPLORE and HORIZON are enrolling patients at sites based in the US, Europe and Australia.

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