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Wednesday, October 20, 2010

[T.S.R:16064] GSK-Italian Allies To Focus On Rare Diseases



GlaxoSmithKline said Monday it formed a new alliance with Fondazione Telethon and Fondazione San Raffaele in a move meant to boost its unit focused on treatments for rare diseases.

Under the deal, Glaxo will pay 10 million euros to Fondazione Telethon, which will be eligible for further unspecified payments upon meeting milestones. Fondazione Telethon and Fondazione San Raffaele are Italy-based organizations focusing on rare genetic disorders.


The partnership will focus on ADA Sever Combined Immune Deficiency, a rare genetic disorder that leaves patients highly vulnerable to infectious diseases.


GlaxoSmithKline ( GSK - news - people ) launched its rare diseases unit in February. It is focusing on Atriance and Arranon, which are potential treatments for a form of leukemia that affects only a few hundred patients worldwide each year.

Marc Dunoyer, global head of the unit, said the U.K.-based company has an established history of successfully researching and developing treatments for rare diseases.


"However, with less than 10 percent of patients with rare diseases currently being treated worldwide, we recognize the size of the challenge, but also the opportunity to deliver new medicines to patients," he said in a statement.


He said the unit will focus on diseases where it can "realistically" make a significant contribution. The unit has adopted a systematic approach, which he said is critical to helping make the right decisions.


"Our aim is to have a list of around 200 priority diseases that the unit will target - this list will change over time as our scientific knowledge and understanding changes," he said.

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