Eye On the Cure Research News

Mar 23, 2019

First Gene Therapy for Dry AMD Moves into Clinical Trial in UK

Gyroscope Therapeutics, a gene-therapy development company in the UK, has launched the first gene therapy clinical trial for the dry form of age-related macular degeneration (AMD).

Audio version:  Gyroscope Therapeutics, a gene-therapy development company in the UK, has launched the first gene therapy clinical trial for the dry form of age-related macular degeneration (AMD). The 10-participant, Phase 1/2 study is being led by Dr. Robert MacLaren, professor of ophthalmology at the University of Oxford, and will be taking place at three locations in the UK.

“There are currently limited treatments for dry AMD, which in many cases will progress to the wet form several years later,” says Dr. MacLaren, who is also the lead investigator for X-linked retinitis pigmentosa and choroideremia gene therapy clinical trials. “We now know a lot about the role of the complement system in AMD and also how to deliver gene therapies safely to the fovea in patients with good visual acuity. Hence it is logical now to explore early preventative interventions that might have an impact in stopping progression of this disease.”

The emerging Gyroscope treatment was developed to slow the progress of dry AMD, thereby preventing or slowing debilitating central vision loss. Therapy developers believe a single subretinal injection of the gene therapy may work for several years or the lifetime of the patient.

Gyroscope’s treatment was designed to inhibit the complement system, part of the innate immune system which helps fight bacteria and other pathogens. However, researchers have learned that the complement system is overactive in people with AMD, leading to degeneration of the macula, the central region of the retina.

AMD is the leading cause of blindness in people over 50 in developed countries. The condition affects approximately 10 million people in the US and 150 million worldwide. Risks for AMD include increasing age and smoking. AMD can also run in families; scientists have identified several genetic variations that can increase or decrease risk for the condition.

AMD begins as the dry form, which is characterized by the accumulation of protein deposits known as drusen underneath the retina. Scientists believe drusen cause overactivation of the complement system. Dry AMD can cause central vision loss over time.

About 15 percent of AMD patients will develop the wet form, which is characterized by the growth of unhealthy, leaky blood vessels underneath the retina that cause more rapid central vision loss. Treatments such as Lucentis® and Eylea®, which are delivered by monthly or as needed ocular injections, can inhibit and mop up the vessels to save and restore vision.

The Foundation Fighting Blindness is currently funding 11 dry AMD research projects, and recently announced The Free Family AMD Research Program, which will fund an additional 10 AMD projects over the next five years.