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Cambridge, GSK to Develop Therapies for Lung/Liver Disorder

Illustration of lungs (Mikael Häggström/Wikimedia Commons)

(Mikael Häggström/Wikimedia Commons)

University of Cambridge in the U.K. will partner with the global pharmaceutical company GlaxoSmithKline to discover and develop new medicines to treat a genetic disease affecting the lungs and liver. The university is expected to receive an upfront payment, and is eligible for milestone payments and royalties under the agreement, but the amounts of those payments was not disclosed.

The disorder studied by the partnership is alpha-1 antitrypsin (A1AT) deficiency, a common genetic condition affecting one in 2,000 people in the U.K. The A1AT protein is produced mainly in the liver and circulates to the lungs, where it serves a protective function against enzymes which can break down lung tissue.

With A1AT deficiency, however, the protein cannot circulate freely and accumulates in the liver, leading to potentially life-threatening liver conditions, including cirrhosis. Without A1AT moving to the lungs, tissue in the lung can also break down, making patients susceptible to early onset emphysema. The only available treatments for the condition are liver transplants for cirrhosis and protein replacement therapy for emphysema.

David Lomas in the Cambridge Institute for Medical Research has conducted studies that define the mechanism that underlies protein accumulation in the livers of individuals with A1AT deficiency. The collaboration with GSK is expected to combine the company’s expertise in drug discovery and development with Lomas’s research to develop treatments. Work on the project will take place both at Cambridge and at GSK labs.

The partnership is part of GSK’s Discovery Partnerships with Academia program that aims to match GSK’s drug discovery and commercialization skills with academic researchers to develop new medicines. GSK says the program offers academic researchers access to GSK resources and financial support, along with the prospect of joint publications with GSK authors.

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