Prosensa

Prosensa is using exon-skipping technology to develop oligonucleotide based therapeutics and correct gene expression in diseases with large unmet medical needs, in particular neuromuscular disorders. The company is developing a medicine for Duchenne Muscular Dystrophy, a lethal childhood genetic disease that has no curative therapy. Prosensa went public in June 2013 and was acquired by BioMarin for up to $840 million in February 2015.