We aim to develop innovative, RNA-based therapeutics to fill unmet medical needs for patients with genetic diseases
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About Prosensa
Prosensa is a biopharmaceutical company focused on 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 disorders.
RNA Modulation
RNA-modulating therapeutics provide a powerful tool for targeted modulation of gene expression. Our unique proprietary technology platform employs single-stranded RNA-based antisense oligonucleotides (AONs) to correct mutated mRNA causing life threatening disorders.
Exon Skipping
Our exon skipping technology is based on interference with exon inclusion signals during the splicing of the pre-mRNA, in order to induce the skipping of the targeted exon from the mature mRNA (messenger RNA) and to restore the level of functional transcripts.