GSK inks $300m rare diseases alliance with Angiochem

The collaboration - which could be worth more than $300 million - will see Angiochem create compounds for LSDs using its engineered-peptide compound (EPiC) technology platform. The peptides will be designed to cross the blood brain barrier and restore enzyme function in the central nervous system.

While there are a number of enzyme replacement therapies on the market for LSDs - for example Genzyme's Cerezyme (imiglucerase) and Shire's Vpriv (velaglucerase alfa) for Gaucher disease - these are unable to enter into the CNS so address only the peripheral symptoms of LSDs.

Central symptoms seen with LSDs can include developmental delay, seizures, dementia deafness and blindness.