ARTICLE | Emerging Company Profile

Ultragenyx: Back to basics

Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx

December 19, 2011 8:00 AM UTC

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...