RNA Modulation

RNA-modulating therapeutics provide a powerful tool for targeted modulation of gene expression. Prosensa’s unique proprietary technology platform employs single-stranded RNA-based antisense oligonucleotides (AONs) to correct mutated mRNA causing life threatening disorders.

In contrast to double-stranded RNA molecules such as RNAi, Prosensa’s AON technology, based on a rational sequence-specific design, offers a subtle, versatile, and allele-specific (re)engineering of RNA processing.

Our AON compounds are designed to:

  • interfere with splicing in order to induce exon skipping, enhance exon inclusion, or correct splicing mutations,
  • remove mutant RNA or protein domains, or
  • block RNA expression.

RNA modulating therapeutics: Exon Skipping, Exon Inclusion, Block RNA expression, Domain Removal, Splicing Correction, Mutant RNA Removal

Overview of therapeutic applications of the Prosensa platform

This broad technology platform facilitates development of therapeutic molecules to a variety of genetic diseases, including neuromuscular disorders such as Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM1), Huntington's Disease (HD) and Spinal Muscular Athrophy (SMA).