RARE

Ultragenyx Pharmaceutical Inc.

A biopharmaceutical company that develops and sells treatments for rare and ultra-rare genetic diseases, many of which had no approved therapy before. Its marketed medicines include CRYSVITA for a bone disorder called X-linked hypophosphatemia and MEPSEVII for a lysosomal storage disease. Founded in 2010 by pediatric geneticist Emil Kakkis, the company's name blends "ultra" (for ultra-rare diseases) with "genyx" (for genetics), and it trades on the stock market under the ticker symbol RARE.