"Call it a mistake--fine." Investors, meanwhile, were getting sick of the company raking in revenue but showing no profit. Bienaimé listened and took cost-cutting measures, including stopping the cancer effort and a second rare-disease program. BioMarin has previously recorded a profit as a result of asset sales but predicts its continuing operations will turn cash-flow-positive next year. The most tantalizing prospect is something BioMarin licensed from University College London and St. Jude Children's Research Hospital: a treatment for hemophilia. It is a gene therapy in which a virus deposits a gene in patients' liver cells that produces a protein missing in the most common type of hemophilia. In a small clinical trial only a few months long, the treatment seems to return patients' protein levels to almost normal. "It really is amazing. It's just a wonderful time," says Timothy Nichols, a hemophilia expert at the UNC School of Medicine who is not paid by BioMarin.