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Case Study:  Quethera

In 2014 Dr Peter Widdowson came across the research of Professor Keith Martin of the Department of Clinical Neurosciences. Professor Martin was working to develop a gene therapy for glaucoma, the leading cause of irreversible blindness worldwide.

Dr Widdowson proposed that they work together to develop and enhance Professor Martin’s original work, combining Dr Widdowson’s pharmaceutical industry experience with Professor Martin’s medical expertise to bring a new therapy to patients. The result was spin-out company Quethera, since acquired by Astellas. The company’s mission was to slow or prevent the loss of sight in patients with glaucoma.

Current glaucoma treatment

Glaucoma-associated blindness is caused by the death of the retinal nerve cells that carry visual signals to the brain. Because these cells cannot regenerate, therapies to protect them are essential. Lowering pressure within the eye using surgery or eye drops is the only treatment proven to reduce the risk of visual loss in glaucoma, but about one in eight patients will still become blind in at least one eye despite pressure-lowering therapy.

Quethera’s solution

Quethera was created in order to pursue a very different approach: putting protective genes into the retinal cells. The delivery mechanism is a recombinant, adenoassociated viral vector system (rAAV). The goal is longterm control of the disease via a single injection. Cambridge Enterprise Seed Funds invested in Quethera in 2015. The money supported pre-clinical development of the therapy and establishment of a strong intellectual property platform. It also allowed Quethera to do research through the University, funding a postdoc in Professor Martin’s lab to test the effectiveness of Quethera’s gene therapy. In March 2016 Quethera won a Wellcome Trust Pathfinder Award to support further testing of the gene therapy constructs in experimental models of glaucoma.

Astellas

When its lead pre-clinical candidate demonstrated significantly improved survival of retinal ganglion cells, Quethera caught the attention of Astellas Pharma, a global pharmaceutical company headquartered in Tokyo. In August they announced plans to acquire the spin-out.

Under the terms of the deal, Astellas could pay as much as £85 million (in upfront and contingent payments) for Quethera, which becomes a wholly-owned subsidiary.

The deal will speed the development of Quethera’s gene therapy construct. With such a high unmet medical need, this is good news for patients who are at risk of losing their sight.

 

I believe the rAAV program has potential as a new therapeutic option for the treatment of refractory glaucoma through an intraocular pressure-independent mechanism.

Dr Kenji Yasukawa. President and CEO, Quethera