Targeting RNA in Drug Development Emerging Tools and Therapeutics 

As scientists have overcome technical hurdles and learned to harness RNA's versatility, its promise as a pharmaceutical agent has grown considerably over the last few years. The fact that RNA is complementary to DNA allows RNA to be used as a blocking agent to prevent the expression of defective genes-antisense technology elegantly exploits this property. Elsewhere, catalytic RNAs (ribozymes) cleave specific RNA targets and in doing so can downregulate or even repair dysfunctional genes. Commercial development of RNA therapeutics targeting a range of conditions from infectious disease to cancer has been led by a cadre of more than 20 companies, most of which are small biotechnology firms. Increasingly, major drug makers and larger biotech players are stepping up investment in RNA-based treatments, as evidenced by the rise in their partnerships with and equity stakes in firms such as Isis Pharmaceuticals and Genta.

Based on interviews with leading experts in the field, this report examines the technological and business trends driving growth in RNA therapeutics. The state-of-the-art in antisense, ribozymes, aptamers, spiegelmers, RNA interference and numerous other current technologies are discussed, as well as the limitations of these technologies. A timeline projecting when RNA-based pharmaceuticals currently undergoing clinical trials can be expected to make their way to market is included, as well as a market forecast and a comprehensive analysis of recent dealmaking activity in RNA therapeutics.

Thought Leaders Interviewed for This Report:

C. Frank Bennett, Isis Pharmaceuticals; Mike Coon, Cell Therapeutics; Christophe J. Echeverri, Cenix BioScience; David Epstein, Archemix Corp.; James Summerton, Gene Tools, LLC; Nassim Usman, Ribozyme Pharmaceuticals, Inc.; Tod Woolf, Sequitur 

Publication date/length: December 2002, 136 pages.

Publisher: Cambridge Healthtech Institute 

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