Gene therapy’s fight against glaucoma

A University of Cambridge gene therapy spin-out is developing a treatment that could prevent the deleterious effects of glaucoma, the leading cause of irreversible blindness worldwide. Quethera was founded in 2013 by Dr Peter Widdowson, who is also Quethera’s CEO. The company builds on the research of co-founder Professor Keith Martin of the Department of Clinical Neurosciences.

In England and Wales, the National Health Service estimates that there are upwards of 500,000 people with glaucoma, with more than 50,000 of these blind in at least one eye due to the condition. A report commissioned in 2003 by the National Institute for Clinical Excellence (NICE) reported that if only 10% of those affected by glaucoma received earlier treatment to slow or prevent the development of visual impairment, it could save the government between £555 million and £1 billion in future costs. Recent estimates predict that by 2020 there will be 11 million people worldwide blind due to the disease.

Glaucoma-associated blindness is caused by the loss of retinal nerve cells that carry visual signals to the brain. These nerve cells cannot regenerate, so it is vital that therapies are sought that can delay or prevent their deterioration. There are currently no licensed therapies to prevent nerve damage: all current therapeutic approaches are aimed at treating ocular hypertension through pressure release in the front of the eye using surgery or eye drops. However, even in populations with access to the most advanced treatments, an estimated 1 in 8 patients will still become blind in at least one eye.

With such a high unmet medical need, Quethera is developing a gene therapy to control the disease long-term via a single injection. While gene therapy is now considered an established technology, Quethera’s intellectual property protects the design of the novel gene construct itself.

The project began in 2014 when Widdowson came across the research of Professor Martin of the Clinical Neurosciences Group at the University of Cambridge and sought collaboration. With Widdowson’s expertise in commercialisation and Martin’s medical background, they pooled their expertise to form Quethera.

In January 2015, Cambridge Enterprise was contacted, and supported the establishment of Quethera with a round of seed funding, alongside Midven’s Rainbow Seed Fund. The funding enabled pre-clinical development of their therapy and the establishment of a strong intellectual property platform. A provisional patent application based on the technology has now been filed with the UK patent office.

The seed investment also allowed Quethera to undertake research through the University of Cambridge, funding a post-doctoral researcher in Martin’s laboratory to work on the design and construction of the gene therapy and to test its effectiveness. Cambridge Enterprise has also provided advisory support and links to venture capital companies with the aim of raising a Series A fund to advance the therapy to clinical trials.

In March 2016, Quethera was awarded a grant from the Wellcome Trust Pathfinder Award Scheme to support the costs of testing the gene therapy constructs and evaluate the effectiveness of individual components in laboratory animals. Current work in Martin’s laboratory is focused on improving the dynamics of the gene therapy coverage across the retinal surface, to optimise the level of protection for the maximal number of retinal cells. Quethera plans to advance the gene therapy into more sophisticated animal models of glaucoma to examine whether it is effective.

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