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Prosensa is a biotechnology company engaged in the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy and Huntington's disease. Prosensa's portfolio of clinical and pre-clinical RNA-based drug candidates is currently focused on the treatment of DMD. Prosensa’s lead product, drisapersen, has completed Phase III clinical trials and a New Drug Application (NDA) has been submitted to the Food and Drug Administration (FDA). Three additional products, PRO044, PRO045 and PRO053 are in clinical development, and PRO052 and PRO055 are in advanced preclinical development. In parallel, Prosensa has also advanced program called PROSPECT into pre-clinical testing, which includes a new and innovative application of its exon-skipping technology platform, applying multiple exon skipping, to specifically target rarer mutations (initially in exon 10 to 30 region) in the dystrophin gene. This approach could have applicability between 5-13% of the DMD population. In addition to its clinical trials, Prosensa is running a Natural History Study, which has completed enrollment with 269 patients across the US, Europe and Latin America. The purpose of this study is to characterize the natural history and progression of DMD, to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible. Prosensa commenced operations in 2002 and is located in Leiden, The Netherlands. The company works closely together with academia, patient groups and experts worldwide. Prosensa entered into an exclusive licensing agreement in 2003 with Leiden University Medical Center (LUMC) for their proprietary RNA modulation exon-skipping technology to develop treatments for DMD, other neuromuscular disorders and indications outside the field of neuromuscular disorders. In November 2014, BioMarin Pharmaceutical agreed to pay up to $840 million to acquire Prosensa.〔http://online.wsj.com/articles/biomarin-to-pay-up-to-840-million-for-prosensa-1416826801〕 ==External links== * 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「Prosensa」の詳細全文を読む スポンサード リンク
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