Trial underway to study safety of retinal gene therapy for dry AMD

Last week, an 80-year-old Oxford woman became the first person to receive an experimental retinal gene therapy for dry AMD. The single-dose treatment, GT005, is designed to calm an overactive complement system in people with a specific genetic variation tied to dry AMD.

Researchers are assessing the therapy’s safety and biological activity in Gyroscope Therapeutics’ phase 1/2 FOCUS trial, which is currently enrolling additional patients with geographic atrophy secondary to AMD.

The trial's inaugural patient has bilateral AMD with central vision loss in her left eye. Robert MacLaren, MD, an ophthalmology professor and vitreoretinal surgeon at the University of Oxford, administered the therapy by detaching the patient’s retina and injecting her eye with a solution containing the viral vector, which is designed to infect retinal pigment epithelium cells and produce an anti-inflammatory protein.

“The idea of this gene therapy is to ‘deactivate’ the complement system, but at a very specific point at the back of the eye, so the patient would otherwise be unaffected by it,” MacLaren says.

Although the trial is primarily focusing on the drug’s safety, investigators are hoping the therapy can preserve the patient’s remaining vision without the need for a repeat procedure. The only gene therapy approved for the eye, Luxturna, has shown it can halt or even reverse vision loss in patients with inherited retinal dystrophy through at least 3 years.