Shire is developing a recombinant enzyme replacement therapy produced in human cell lines, currently in phase III. They partnered with Amicus in order to develop the oral chaperone Plicera, which works in a different way (by binding to the mutant enzyme to enhance trafficking to the lysosome). Results from Plicera's 6-month phase II study, are expected in 3Q08. Plicera is potentially a less expensive and more convenient therapy than ERT, and may have the advantage of crossing the BBB and treating Types 2 and 3 patients.
Shire obviously believes in the chaperone approach as they teamed up with Amicus on another one - Amigal for Fabry disease.
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