GSK Continues Emphasis On Rare Diseases With Pact For "Bubble Boy" Gene Therapy
This article was originally published in The Pink Sheet Daily
Recently launched GSK rare diseases unit will focus on four primary therapeutic areas.
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In-licensing and development of so-called "bio-betters" will play critical roles in Pfizer's move, announced June 14, into the rare diseases space. The Rare Diseases Research Unit, to be overseen by Jose Carlos Gutierrez-Ramos, senior VP, Biotherapeutics Research and Development, will be part of Pfizer's worldwide R&D unit devoted to discovering novel medicines for orphan diseases, those affecting fewer than 200,000 patients. Initially it will target hemophilia and muscular dystrophy, both caused by genetic mutations
Under the deal, which has all the hallmarks of a GSK option-based alliance, Isis gets $35 million upfront, and will deliver five drugs through PoC.
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