Orphans by Algorithm

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...