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...