Realizing the Promise of Therapeutic RNAi: Emerging Strategies for RNAi Delivery
Decision Resources
January 31, 2008 26 Pages - SKU: DECR1682308
|
|
The discovery of RNA interference (RNAi) has renewed interest in developing nucleic acid drugs based on
targeting messenger RNAs (mRNAs). This field 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 field,
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.
Please note, the PDF e-mail from publisher version of this report is for a global site license.
|
- 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
Share this report
Other tasks Related Markets Antisense & RNAi Technology Reports Free Alert Me service Receive bi-weekly email alerts on new market research Sign Up Today!
|
