When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, he says pharmaceutical
companies wouldn’t give him the time of day. The treatment was eventually developed and marketed as Aldurazyme by BioMarin and its partner Genzyme.
Kakkis, who eventually became chief medical officer of BioMarin, has just raised $45 million for UltraGenyx, a new company he’s formed to develop treatments for rare diseases. We spoke to Kakkis about his ambitious plans for Ultragenyx, why he thinks he can produce 10 drugs in 10 years, and why Big Pharma is suddenly interested in rare disease.