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Progress in RNA-Based Therapeutics
Decision Resources, Inc, Aug 2005, Pages: 19
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 we focus on three such technologies in this report: antisense oligonucleotides, RNA interference, and microRNAs. These three technologies are particularly dynamic; antisense, for example, has suffered a series of negative clinical trial results that may have stalled its progress, and RNA interference has been hailed as a revolutionary breakthrough and has generated interest from more than 100 companies.
In this report, we describe these three RNA-based technologies, review recent progress in RNA-based drug discovery and development, and profile the activities of companies in the field.
Business Implications
- RNA-based technologies include antisense oligonucleotides (ASOs), RNA interference (RNAi), and microRNA (miRNA). Companies have been testing ASOs in the clinic for ten years, and one antisense product, fomivirsen (Isis Pharmaceuticals’ Vitravene), is on the market. RNAi is a new, hot technology that is having a major impact on drug discovery and development. The newly emerging technology of miRNAs may have therapeutically useful links to cancer and other diseases.
- Significant advances in RNAi-based drug development have occurred in recent months, including the demonstration of
proof-of-principle and in vivo efficacy of RNAi products in several animal models. 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. Although RNAi-based molecules could well become a powerful new class of therapeutics, the clinical potential of RNAi products remains uncertain.
- Antisense-based therapeutics stumbled in 2004; several Phase III clinical trials produced discouraging results. Some antisense companies may be able to survive through clinical trials, but obtaining funding for these trials will be difficult unless significant positive results are demonstrated. Antisense therapeutics may eventually achieve real clinical success, but the path to this goal is still unknown.
- Significant technological challenges remain for the development of RNA-based therapeutics. They include compound stability, unwanted side effects, specificity, and delivery. Researchers are working in all of these areas to overcome these challenges. In particular, effective delivery methods will continue to be developed in parallel with clinical trials that assess the safety and efficacy of ASOs and RNAi-based products.
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