A Rare Disease Pioneer Scores $45 Million for New Company

News | 06. 26. 2011

Ultragenyx Pharmaceutical

When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, a treatment for the rare and fatal lysosomal storage disorder MPS1, he says pharmaceutical companies wouldn’t give him the time of day, let alone funding. The treatment was eventually developed and marketed as Aldurazyme by BioMarin and its partner Genzyme. Kakkis went on to serve in various positions at BioMarin over 11 years including chief medical officer. But he’s just raised $45 million for UltraGenyx, a new company he 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.