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Significant Technological Challenges Remain for the Development of RNA-Based Therapeutics

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Research and Markets has announced the addition of Progress in RNA-Based Therapeutics to their offering.

The field of nucleic acid-based therapeutics ranges from gene therapy technologies for replacing defective or missing genes to gene-blocking or gene-silencing technologies for preventing the expression of deleterious genes.

RNA-based therapeutics are a relatively new addition to the field, and this report focuses on three such technologies in this report: antisense oligonucleotides, RNA interference, and microRNAs.

In this Decision Resources report, these three RNA-based technologies are described, recent progress in RNA-based drug discovery and development is reviewed, and the activities of companies in the field are profiled.

RNA-based technologies include antisense oligonucleotides, RNA interference, and microRNA. Companies have been testing ASOs in the clinic for ten years, and one antisense product, fomivirsen (Isis Pharmaceuticals' Vitravene), is on the market.

Acuity Pharmaceuticals and Sirna Therapeutics initiated the first two human clinical trials of RNAi-based products in 2004; additional clinical trials are anticipated in 2005 and 2006.