Gyroscope Therapeutics announces first patient dosed in Phase I/II FOCUS study in dry AMD

Stevenage, 19 February 2019 – Gyroscope Therapeutics (Gyroscope), an ophthalmology company developing genetically defined therapies for retinal diseases, has commenced dosing in a Phase I/II clinical trial in dry age-related macular degeneration (AMD). The FOCUS study is an open label, dose escalation, multicentre study to assess the safety and biological activity of Gyroscope’s novel therapeutic approach (GT005) in patients with geographic atrophy due to dry AMD. The trial successfully dosed the first patient in January and is currently enrolling patients in the UK. An account of the ground-breaking treatment was the subject of a recent BBC News broadcast. 

AMD is one of the leading causes of blindness worldwide. By the year 2020, an estimated 196 million people globally will have AMD and 11 million will have significant vision loss. Dry AMD, which accounts for the majority of all AMD, is a slow deterioration of the cells of the macula which can lead to irreversible loss of visual function and eventual blindness. There are no approved treatments for the dry form of AMD.

Gyroscope is leveraging knowledge of the genetic factors associated with advanced AMD and the inflammatory biology that drives progression of the disease. The Company has leveraged its gene therapy and surgical platform to develop deliver an endogenous anti-inflammatory protein to the retina of patients. The goal is to develop a one-time treatment for the disease. The objective of the FOCUS study is to assess the safety of Gyroscope’s innovative gene therapy approach and record early indicators of biological activity in this devastating condition. Treatment of the first patient was carried out by Robert MacLaren, Professor of Ophthalmology at the University of Oxford, UK.

Dr Soraya Bekkali, Chief Executive Officer of Gyroscope Therapeutics, commented: “Our goal at Gyroscope is to advance new therapies for the treatment of debilitating eye diseases such as agerelated 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. We are delighted to have dosed the first patient in the FOCUS study. We believe this is a great step forward in developing a therapy to treat dry AMD, while we continue our efforts on expanding our clinical programmes internationally.”

Robert MacLaren, Professor of Ophthalmology, University of Oxford added: “AMD is the number one cause of untreatable blindness in the developed world. A genetically defined treatment administered early on to preserve vision in patients who would otherwise lose their sight would be a tremendous breakthrough and certainly something I hope to see in the near future.”

Chris Hollowood, Chief Investment Officer of Syncona and Chairman of Gyroscope, said: “The evolution of Gyroscope into a clinical stage company is a great milestone and an example of Syncona’s expertise in harnessing ‘Third Wave’ technologies to develop therapies for serious diseases. Gene therapies are at the forefront of a new generation of treatments for retinal diseases and we are excited by the potential of Gyroscope’s novel approach to address one of the world’s biggest causes of blindness.”

About Gyroscope: vision for life

Gyroscope Therapeutics is developing cutting edge, genetically defined therapies for the treatment of eye diseases linked to an unbalanced complement system, a part of the immune system. The Company was founded to capitalise on a convergence of advancements made in the understanding of the complement system’s impact on eye disease, the genetic basis of Agerelated Macular Degeneration (AMD) and gene therapy as a mode of treatment delivery.

Gyroscope’s lead product, GT005 (the FOCUS study), is a novel retinal gene therapy aimed at delivering a targeted, one-time treatment to genetically defined patients with advanced dry AMD. GT005 aims to restore the balance of a complement system known to be hyperactivated in AMD. Beyond GT005, Gyroscope is building a pipeline of gene therapy products for the treatment of eye diseases with high unmet medical need.

Gyroscope is a private company headquartered in Stevenage, UK and is supported by experts in complement biology, AMD and gene therapy. Investors include Syncona Ltd. and Cambridge Enterprise. Further information can be found at

About Syncona

Syncona is a leading FTSE250 healthcare company focused on founding, building and funding global leaders in life science. Our vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders. We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses. We take a long-term view, underpinned by a deep pool of capital, and are established leaders in gene and cell therapy. We focus on delivering dramatic efficacy for patients in areas of high unmet need. Further information can be found at

About Retinal Gene Therapy in Age-related Macular Degeneration (AMD)

Gene therapy involves the insertion of genes into cells and uses the cell machinery to produce a protein that is either missing in the body or dysfunctional. Treatment with retinal gene therapy involves an operation to detach the retina and inject a solution containing a virus underneath. The virus contains a modified DNA sequence, which infects cells, called the retinal pigment epithelium (RPE), and corrects the genetic defect that causes AMD. If successful, gene therapy would only need to be performed once, as the effects are believed to be long-lasting.

About Age-related Macular Degeneration (AMD)

AMD is the most common cause of blindness among the elderly in the industrialised world, affecting more than 35 million people in the western world alone. The frequency of the disease increases significantly with age, with more than 10% of the population over 70 years old showing signs of AMD. It presents as a progressive and debilitating loss of vision in the centre of the visual field (macula). As the disease progresses to the atrophic form, also called Geographic Atrophy, it becomes increasingly difficult for patients to recognise faces, drive, read, or perform other activities of daily life. It can also make it more difficult to see contrast and can change the way colour is seen.