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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 building blocks called nucleotides (A,C,U,G). Proteins are made from 20 different building blocks called amino acids.

  • RNA-based drug discovery is a rational and efficient process. Isis' antisense drugs 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, oligonucleotide medicinal chemistry, formulations and RNase H, RNAi, and splicing mechanisms. Isis has also applied the principals of its oligonucleotide chemistry to develop other drugs targeting RNA, including microRNAs, an emerging new area of biology.  In order to fully explore the therapeutic opportunities in microRNAs, Isis and Alnylam Pharmaceuticals established Regulus Therapeutics, a company focused solely on the discovery, development and commercialization of microRNA therapeutics.

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 Regulus Web site to learn more about microRNAs and the therapeutic potential of microRNA-based therapeutics. 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.

 

Author: Timothy A. Vickers, Seongjoon Koo, C. Frank Bennett, Stanley T. Crooke, Nicholas M. Dean and Brenda F. Baker
Research article tiltle: Efficient Reduction of Target RNAs by Small Interfering RNA and RNase H-dependent Antisense Agents. read more >