From LSD To GD To PD

Multiple companies are currently trying to find new and better ways to treat inherited lysosomal storage disorders (LSD), which are responsible for more than fifty rare and orphan diseases. Although Type 1 GD is the most common LSD and doesn’t generally involve the central nervous system (CNS), most LSDs do, including Hunter syndrome (MPS II), Tay Sachs disease, and Niemann-Pick disease, type C. Several companies are looking for new ways to treat LSD symptoms in the CNS, and, if possible, to find ways to extend the agents to PD. Among them, ArmaGen Technologies Inc. uses its protein-fusion technology to get large molecule agents into the brain that otherwise would not transit the blood-brain barrier. ArmaGen’s lead drug candidates focus on CNS therapies for LSDs that include MPS I and II, but it also has an early-stage agent in its pipeline, AGT-190, that targets PD. Deploying a related “Trojan horse” platform technology for getting large molecules into the brain, Angiochem Inc. struck a deal in 2012 potentially worth more than $300 million with $31.5 million in up-front cash, with GlaxoSmithKline PLC, for LSD-targeting fusion agents that could reach into the brain. Although the partnership is focused on rare LSDs, it may, according to news reports, eventually extend to PD. [See Deal]

A few smaller companies have set their sights on PD. Three are using synuclein-antibody strategies as active or passive vaccines:...