Prosensa was 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 was acquired by BioMarin.[1]
History up to the sale
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.
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 (in June 2015) a New Drug Application (NDA) has been accepted by the US FDA.[2]