Abstract
Introduction:
The discovery of RNA interference (RNAi) has renewed interest in developing
nucleic acid drugs based on targeting messenger RNAs (mRNAs). This fi eld of
research offers the potential to produce low-dose, nontoxic RNAi agents to
treat important diseases. Venture capitalists have invested large sums of
money in RNAi companies, and Big Pharma is acquiring and partnering with RNAi
specialty companies. The success of RNAi therapeutics rests on the development
of technologies that can deliver these agents to their targets.
Questions Answered in This Spectrum Report:
- Several companies are focusing on RNAi-based therapeutics. Which are
the leading companies in the fi eld, and which ones are in partnership with or
have been acquired by Big Pharma? What projects are they undertaking?
- The holy grail for RNAi drug companies remains systemic delivery.
Companies are competing furiously to develop viable systemic delivery methods.
What are the promising technologies for systemic delivery, and which
companies are at the forefront of delivery technology?
- Opko Health' s bevasiranib is the most advanced RNAi therapeutic. It is in
Phase III clinical trials for wet age-related macular degeneration. What
other RNAi therapeutics have been developed and for what disease(s)? At what
stage of development are they?
Scope:
- SRNAi: Its mechanism of action.
- Delivery of RNAi therapeutics: Direct delivery of naked siRNA,
systemic delivery of siRNA using stable nucleic acid lipid particles (SNALPs),
lipidoid nanoparticles, siRNA-lipoplexes, interfering nanoparticles, TargeTran
nanoparticles, covalent conjugation of siRNA to a targeting molecule,
antibody-mediated targeting of siRNAs, systemic targeting of siRNAs to the CNS.
- Top companies in RNAi research: Alnylam Pharmaceuticals, Sirna
Therapeutics, RXi Pharmaceuticals, Silence Therapeutics.
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