Isis is establishing a new paradigm for drug discovery: binding, or hybridizing, drugs to RNA instead of proteins. RNA represents a new drug receptor, as the focus of the pharmaceutical industry for more than 100 years has been on discovering drugs that bind to proteins.
RNA is an optimal drug target for several reasons:
RNA is universal. It is vital to all living things, as it affects the expression of all genes.
RNA is a simpler molecule than proteins in that it is composed of four fundamental molecules called nucleotides (A,C,T,G). Proteins are made from 20 different molecules called amino acids.
RNA-based drug discovery is a rational and efficient process. Isis' antisense compounds are designed to interact precisely with a specific sequence of RNA.
RNA is a validated target for drug discovery. The world’s first antisense drug, Vitravene® (formivirsen), has been successfully commercialized. In addition, small molecule antibacterial drugs, which are now understood to work by binding to RNA, are also marketed.
Isis has led the industry in the development of technologies to exploit RNA. The Company is best known for its pioneering work in the development and understanding of antisense technology including biology, chemistries, formulations and RNase H, RNAi, and splicing mechanisms. Additionally, Ibis Biosciences, Inc., Isis' majority-owned subsidiary, has invented a new platform for discovering small molecule drugs that bind to RNA and has expanded the application of this technology to the creation of a revolutionary biosensor system.
In this section, there is information about antisense research that ranges from basic biology to a review of the latest therapeutic advancements in antisense drug discovery. Additionally, please visit the Ibis website to learn about the Ibis T5000 Biosensor System, a revolutionary system for the identification of infectious organisms. In the process of developing these technologies, Isis scientists have made a wide range of discoveries and inventions, which the Company has protected in its large patent estate. Read about Isis’ leading intellectual property portfolio in RNA-based drug discovery in this section as well.