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15 March, 2019 - 09:54 By Kate Sweeney

Cambridge key to world-first gene therapy for cause of blindness

Dr Robert Tansley

Cambridge Innovation Capital and Cambridge Enterprise, the university’s commercialisation arm, are key investors in a Herts-based startup pioneering the use of genetics to cure one of the world’s biggest causes of blindness.

The first patient with age-related macular degeneration (AMD) has already been treated by Stevenage business Gyroscope Therapeutics which has received significant but undisclosed financial backing led by quoted UK healthcare company Syncona.

The technology is exciting medics specialising in eye disease all over the world.

Gyroscope is developing cutting edge, genetically defined therapies for the treatment of eye diseases linked to an unbalanced complement system, which is a part of the immune system.

A significant portion of Gyroscope’s founding IP came from the laboratory of Professor Sir Peter Lachmann at the University of Cambridge. This was developed, licensed and invested in by Cambridge Enterprise, the commercialisation arm of the university. CIC is underpinning the venture long term via its patient capital model.

As well as Professor Lachmann’s work, Gyroscope further leveraged the research of David Kavanagh from Newcastle University and Andrew Lotery at the University of Southampton to identify the patient population that would benefit from the company’s products.

Gyroscope recently announced that the first patient had been successfully dosed in a Phase I/II clinical trial to assess the safety and biological activity of lead product, GT0005, which uses a novel therapeutic approach in AMD. 

This approach leverages knowledge of the genetic factors associated with advanced AMD and the biology of the inflammation that drives progression of the disease. 

Gyroscope’s gene therapy and surgical platform delivers an endogenous anti-inflammatory protein to the retina of patients. The goal is to develop a one-time treatment for the disease.

AMD is one of the leading causes of blindness. By the year 2020, an estimated 196 million people globally will have AMD and 11 million will have significant vision loss. 

Dry AMD, which is the most common form of 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 currently no approved treatments for dry AMD.

Dr Robert Tansley, operating partner at CIC, said: “The team at Gyroscope has developed an exciting and pioneering approach which aims to tackle a range of eye diseases with significant unmet medical need. 

“While the clinical studies are very early stage and the treatment route is novel, the potential for these therapies, if successful, is enormous. As such, Gyroscope represents a great addition to the CIC portfolio and we look forward to supporting Soraya and her team as the business grows and develops.”

Gyroscope CEO Dr Soraya Bekkali explained: “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 programme 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 to expand our clinical programmes internationally.”

• PHOTOGRAPH: Dr Robert Tansley, operating partner at Cambridge Innovation Capital

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