Aug 2007

Shire Human Genetic Therapies (Shire HGT), a business unit of Shire plc, a global specialty pharmaceutical company, is focused on developing innovative therapies for life-threatening genetic diseases.  Shire HGT applies its proprietary technologies, including human cell line, gene activation and Formylglycine Generating Enzyme (FGE), to the discovery, development and manufacturing of protein therapies to treat genetic diseases.  With over 600 employees in 16 countries around the globe, Shire HGT is dedicated to providing hope for patients and their families, for better, healthier lives.

Shire HGT is developing an investigational ERT to replace the deficient or absent arylsulfatase A (ASA) for the treatment of Metachromatic Leukodystrophy (MLD).  The company is using its proprietary FGE technology to create highly productive human cell lines for the manufacture of sulfatases.  Co-expression of FGE with a sulfatase leads to significantly increased enzymatic activity, in vitro, as compared to sulfatases produced without this technology.   Shire HGT’s MLD program is currently in preclinical development.  A natural history study and patient registry are currently in the planning stages, with the goal of beginning patient enrollment by the end of 2007.  These studies will help scientists to better understand disease progression and pathology in MLD, and will be used to inform the design of future clinical trials.

For more information about the program, please contact Carol Cannon at Shire HGT Medical Affairs: ccannon@shire.com