RNAi Therapeutics: Principles, Prospects and Challenges

What Is RNAi...??

RNA interference is a biological method in which molecules of RNA inhibit gene expression by neutralizing the targeted mRNA molecules. This method plays vital role in defending cells against virus. This technique is an innovative method which into the clinical trial phase, and various companies are seeking for the FDA and EU commission approval for their products and technologies. The RNAi therapeutic are formulated to treat diseases such as cancer, haemophilia, age related diseases, and others.

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ncbi.nlm.nih.gov

The discovery of RNA interference less than a decade ago was a turning point for molecular biology. RNAi has become a powerful tool for studies of gene function in mammals. It provides the researcher with the ability to silence virtually any gene with artificial triggers of RNAi and utilizing the cellular machinery for efficient targeting of complementary transcripts. Proof-of-principle studies in vivo have clearly demonstrated that both viral and non-viral delivery methods can provide selective and potent target gene suppression without any clear toxic effects. Translational research using breakthroughs in basic RNAi research has taken place with an unprecedented speed and already there are several RNAi based human clinical trials in progress. Despite excitement from a large number of animal model studies, including systemic delivery to non-human primates, there are a number of hurdles and concerns that must be overcome before RNAi will be harnessed as a new therapeutic modality. These include off-target effects, triggering of type I interferon responses, competition with cellular RNAi components and effective delivery in vivo. As with all biological discoveries, understanding of the mechanism is paramount to effective applications in human disease. Given the pace of new findings and discoveries of applications, we anticipate that RNAi will be a major therapeutic modality within the next several years. Hopefully RNAi will revolutionize the treatment of human disease in the same way that it has revolutionized basic research.

Key 10 companies and development in past five years.

Alnylam Pharmaceuticals, Inc., Sanofi, OLIX PHARMACEUTICALS, GlaxoSmithKline plc, Benitec Biopharma, Arbutus Biopharma Corporation, Silence Therapeutics, Rexahn Pharmaceuticals, Inc, F. Hoffmann-La Roche Ltd, Quark

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Reference

  • theinsightpartners.com
  • ncbi.nlm.nih.gov