The oligonucleotide therapies arena has seen a huge upsurge in both R&D and investment activity in recent years. This Scrip analysis explores the science, the companies, and their potential for success.
Key topics covered:
- The story of RNA interference (RNAi); the RNAi process and its therapeutic potential - RNAi advantages: pharmacokinetics; faster drug development? - RNAi disadvantages: delivery and longevity; off-target effects; interactions - micro RNA - Antisense “more rational” than other drug discovery - The Human Genome Project and alternative splicing - Oligonucleotide aptamers - Ribozymes - TLR9 agonists and other immunostimulatory therapeutics - Decoy RNAs - The pharmaceutical players: active companies, both large and small - Therapeutic potential of RNAi in cancer and HIV/AIDS
