Prosensa

Prosensa's lead product candidate was drisapersen, an investigational drug designed to treat a specific subset of DMD patients. The company's therapeutic approach, known as exon skipping, aimed to correct the genetic defect that causes DMD by producing a shorter but functional version of the dystrophin protein, which is essential for muscle function. This approach positioned Prosensa at the forefront of genetic medicine for rare diseases. The business model revolved around extensive research and development, clinical trials, and strategic partnerships. A significant partnership was established with GlaxoSmithKline (GSK) for the development and commercialization of its DMD portfolio; however, this collaboration was terminated in 2014 following disappointing clinical trial results for drisapersen.

Despite the clinical setbacks, Prosensa's expertise and intellectual property in RNA therapeutics remained valuable. The company had a pipeline of other drug candidates for DMD and other rare diseases. In a significant milestone for the company and a notable event in the biotech industry, Prosensa was acquired by BioMarin Pharmaceutical Inc. in November 2014. The acquisition, valued at approximately $840 million, provided BioMarin with Prosensa's portfolio of drug candidates for rare genetic diseases, including drisapersen.