Biotech Values

[jq1234]

In addition to the Orphan drug period they need to develop enzymes that are at least as good (just ask John Crowley or Henri Termir about the difficulties of doing so in Pompe). And now companies are trying to improve on the ERT's. Genzyme has an oral Gaucher program, Amicus is co-administering their chaperone with ERT, Biomarin is attempting approach to improve the binding,

Difficulty in developing ERT is a plus not minus, it limits potential players in the field. If you succeed in one, you eventually succeed in others. Both GENZ, Shire, BMRN have showed that. There is no reason for PLX to succeed in Gaucher only.

Amicus's chaperone as monotherpy in Gaucher failed. It has long way to go for combination with ERT, currently in pre-clinical.

Their cheap/high capacity technology could be a plus too though to me that isn't enough by itself unless their goal is to be a high end "generics" play.

I think that is their current goal. Look at their programs, Gaucher, Fabry, biosimilar Enbrel.