Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx
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.