Realizing the Promise of Therapeutic RNAi: Emerging Strategies for RNAi Delivery

Realizing the Promise of Therapeutic RNAi: Emerging Strategies for RNAi Delivery published by Decision Resources, Inc. in January, 2008. This report consists of 26 Pages and the price starts from US $ 2250.

Introduction

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.

Table of Contents

• Introduction
• Mechanism of RNA Interference
• Design of RNAi Research Reagents and Therapeutics
• Delivery of RNAi Therapeutics
  • Alnylam' s Direct Delivery of Naked siRNA
  • Nastech Pharmaceutical' s Carrier-Mediated Technology
  • Systemic Delivery of siRNA Using SNALPs
    • Alnylam Pharmaceuticals
    • Sirna Therapeutics
    • Intellectual Property Issues
  • Alnylam Pharmaceuticals/MIT' s Lipidoid Nanoparticles
  • Alnylam' s Clinical Progress
  • Silence Therapeutics' siRNA-Lipoplexes
  • RXi' s Interfering Nanoparticles
  • Intradigm' s TargeTran Nanoparticles
  • Covalent Conjugation of siRNA to a Targeting Molecule
    • Alnylam Pharmaceuticals
    • Duke University
  • Antibody-Mediated Targeting of siRNAs
  • Systemic Targeting of siRNAs to the Central Nervous System
• Company Highlights
  • Alnylam Pharmaceuticals
  • Sirna Therapeutics
  • RXi Pharmaceuticals
  • Silence Therapeutics
• Outlook for the Delivery of RNAi Therapeutics

Tables:

• 1. Select RNAi Therapeutics in Development
• 2. Select Technologies for Systemic Delivery of siRNAs
• 3. Select Companies Developing RNAi Therapeutics and/or Delivery Technologies

Figures:

• 1. Mechanism of RNA Interference
• 2. Structure of a Stable Nucleic Acid Lipid Particle (SNALP)
• 3. Schematic of Intradigm' s TargeTran Nanoparticles
• 4. Delivery of siRNAs to Neurons via the Acetylcholine Receptor

Related Reports