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Nov 01, 2010
 |  BioCentury  |  Strategy

Orphans by Algorithm

GSK rare disease strategy includes deals with Amicus and Fondazione Telethon

GlaxoSmithKline plc is taking a stepwise approach to building its new rare diseases unit that for now is relying heavily on external collaborations and in-licensing as a source of late-stage compounds. Last week, GSK licensed exclusive rights to Amigal migalastat from Amicus Therapeutics Inc. to treat Fabry's disease, while earlier in October, the pharma licensed rights to an ex vivo stem cell gene therapy from Fondazione Telethon and Fondazione San Raffaele.

The new unit, which was formed in February, is headed by Marc Dunoyer, who had been GSK's president of Asia Pacific and chairman of Japan. Dunoyer estimates there are 6,000-8,000 rare diseases, but GSK has developed an algorithm to winnow that down to 200 target diseases.

The algorithm takes into account a variety of factors to provide a scientific rationale for targeting a given disease, with the three main criteria consisting of disease prevalence, disease burden and potential for disease modification.

The pharma's target diseases fall into four main therapeutic areas: metabolism and inherited disorders, such as Hunter's syndrome, Fabry's disease or Gaucher's disease; CNS and muscle disorders, such as Duchenne muscular dystrophy (DMD); immunoinflammation, such as graft-versus-host disease (GvHD); and rare malignancies and hematology.

While these specific examples may appear...

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