FOCUS: A study to test a new treatment for dry AMD

Research summary

Age-related Macular Degeneration (AMD) is the most common cause of blindness among the elderly in the industrialised world- affecting more than 35 million people globally. AMD presents as a progressive loss of vision in the centre of the visual field resulting in a blurred area or blank spot in the centre of vision. With ageing, AMD progresses to an advanced stage becoming increasingly difficult for patients to recognise faces, drive, read and/or perform other activities of daily life. The overall UK prevalence of AMD is 2.4%, with a sharp age related increase in occurrence of 4.8% of people over 65 and as many as 12.2% over 80 years of age. This is an open label first in human phase 1/2 study recruiting patients with confirmed AMD. The overall objective is to evaluate the safety, dose response and efficacy of a gene therapy (GT005) in AMD patients. The study will be conducted in three parts; Part 1: dose-escalation, Part 2: dose expansion, and Part 3 dose-escalation with Orbit Subretinal Delivery System(SDS). Parts 1 and 2 will deliver GT005 via the standard transvitreal procedure and Part 3 will deliver GT005 via the Orbit SDS device. The study will be conducted at approximately 10 study sites; surgical procedures will be carried out at one or more central surgical site where the treatment is administered as a single subretinal injection into one eye. Total study length is estimated to be 5 years, including a subject recruitment period estimated at 18 months and a 4 year post-treatment follow-up period.

Animation showing progression of atrophy over a 6 year time period.