We here at FOTF holiday headquarters (not pictured above) can't think of a better Hannukwanzamas present than a treatment for a rare disease whose patients previously had few or no options to help them. One of the big stories this year in our neck of the woods is that many in the biopharma industry are thinking the same thing, too. Of course, motivations for some are fueled in part by premium pricing and favorable regulatory pathways, terms that don't exactly invoke the holiday spirit. But if remedies come to market, we won't quibble. Capitalism is the worst of all pharmaco-economic systems except for all the others.
We've already noted several fundings, including the new companies Ultragenyx and Orphazyme, in this column this year; in the upcoming issue of START-UP, the Capital Matters team will examine a new NIH program, dubbed Therapeutics for Rare and Neglected Diseases, or TRND, that helps both academics and biotechs bring rare-disease programs across the valley of death. TRND has already helped push treatments for sickle-cell anemia and relapsed chronic lymphocytic leukemia into the clinic, with hopes for two more clinical programs in 2012. (TRND's full roster of programs is here.)