Curzion Pharmaceuticals Acquired by Horizon Therapeutics

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.