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

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