British biotech Gyroscope Therapeutics may have stumbled onto a gold mine if its new gene therapy for the most common form of blindness proves effective.
The company is currently treating the first patient in a phase I/II trial of its gene therapy for dry age-related macular degeneration, one of the leading causes of blindness worldwide.
GT005 is a novel retinal gene therapy aimed at delivering a targeted, one-time treamtent to genetically defined patients with advanced Dry AMD that aims to restore the balance of a complement system known to be hyperactivated in AMD.
The drug has been injected into the back of a patient’s eye in an attempt to prevent further degeneration fo the photoreceptor cells in the retina. The patient herself is an 80-year-old woman with geographic atrophy which causes blurring in the central vision of her left eye.
Dr. Soraya Bekkali, CEO at Gyroscope Therapeutics, said: “Our goal is to advance new therapies for the treatment of debilitating eye diseases such as age-related macular degeneration. Building on the research of Gyroscope’s scientific founders, we have been working relentlessly over the last two years to advance our first drug development program into the clinic”
Eye diseases have been a quick target for gene therapies because delivery methods into the eye are rather simple compared to other parts of the body. The first gene therapy approved was Spark Therapeutics’ Luxturna (voretigene neparvovec) for inherited retinal dystrophy caused by RPE65 gene mutations – a treatment that was also cleared in the EU at the end of last year.
By 2020 it is estimated that 196m people will have the disease with 11m already having significant vision loss. Patient figures for the condition in the U.K. number around the 600,000 mark.