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Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx

By Jennifer Rhodes
Staff Writer
Published on Monday, December 19, 2011

Ultragenyx Pharmaceutical Inc. is taking a traditional approach to Orphan Drugs by in-licensing development-ready substrate and enzyme replacement therapies that have a clear mechanistic rationale and proof of concept in animals for rare and ultra-rare genetic diseases.

Founder, President and CEO Emil Kakkis believes there are a few hundred such diseases with no current treatments.