Françoise de Craecker

Horizon Pharmaceutical LLC engages in the development of transformative treatments for people affected by rare and debilitating diseases. It offers PROCYSBI, a delayed-release capsule, which is used for the management of nephropathic cystinosis in adults and for six years and older children. The company was founded by Christopher M. Starr and Todd C. Zankel on April 29, 2002 and is headquartered in Novato, CA.

Novartis Gene Therapies, Inc. developed gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases. The firm’s robust AAV-based pipeline is used to provide advance treatments for Rett syndrome and Friedreich’s ataxia. The company was founded by John D. Harkey, Jr., David G. Genecov and John A. Carbona on March 8, 2010 and was headquartered in Bannockburn, IL.

Part of Amgen, Inc., Raptor Pharmaceuticals Europe BV is a Dutch company that engages in research and experimental development in other physical and natural sciences. The company is based in Louveciennes, the Netherlands.

Transkaryotic Therapies, Inc. (TKT) was a biopharmaceutical company with a major focus on developing products for the treatment of rare diseases. The company is used to markets one product, Replagal™, for the treatment of Fabry disease in the European Union and other countries, and was developing products for Hunter syndrome and Gaucher disease. The company was located in Cambridge, MA.