AMT HOLDING

AMT (Amsterdam Molecular Therapeutics) specializes in the development of gene therapy products intended for treating serious, often rare, diseases with a major medical need. The group's products are based on introducing genes into target organs and tissues in order to accomplish the long-term expression of the genes and correct the genetic defects causing the disease. AMT has a portfolio of 5 products: 3 in late stage pre-clinical development (for treating Parkinson's Disease, Duchenne Muscular Dystrophy and Porphyria), 1 in the Phase I/II clinical study (for treating Hemophilia B) and 1 in the marketing phase (Glybera® for treating lipoprotein lipase deficiency).