Stepping across the barrier
GlaxoSmithKline (GSK) is further strengthening its activity in the area of rare diseases. The London-based pharma giant has signed an early-stage deal to develop and commercialise treatments for lysosomal storage disorders (LSDs). To that end, it has signed a global collaboration with Canadian biotech company Angiochem for upwards of US$300m (€223.7m) to help GSK develop blood-brain barrier (BBB)-crossing enzyme replacement therapies that could for the first time address the CNS symptoms of such diseases. The deal will see Montreal-based Angiochem, which specialises in getting peptide compound drugs past the BBB, to create and develop at least one product for a specified LSD. GSK will assume all development and commercialisation rights to the product. According to Angiochem CEO Jean-Paul Castaign, around 50 rare LSDs have been described so far, and about 20 have central nervous system symptoms that are left unaddressed by current therapies. These are the symptoms that the two companies hope to be able to treat for the first time, along with the peripheral symptoms that to a certain extent can be treated with existing therapies. Angiochem is also developing brain anti-cancer drugs in partnership with Geron.