Introduction
Ultragenyx Pharmaceuticals (RARE) is a mid-cap ($2.46B) biopharma creating and developing innovative therapeutics for ultra-rare and/or orphan as well as genetic diseases. CEO Dr. Emil Kakkis has had a lifelong research career in developing novel therapeutics for rare diseases starting with the approval of two therapeutics for Mucopolysaccharidosis ((MPS)) VI and Phenylketonuria while at BioMarin (BMRN).
Surely, Dr. Kakkis did "put his money where his mouth is" with the creation of the Kakkis EveryLife Foundation in 2009 to accelerate biotech innovation for rare diseases, and in 2010, Ultragenyx was founded.