RNA Therapy: The Next Big Thing after Monoclonal Antibodies?
- Published: October 2008
Introduction
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 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
READ MORE >
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
SPECTRUM Drug Discovery and Design 1-3
RNAi Therapeutics Delivery
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
- Acuity Pharmaceuticals
- Alnylam Pharmaceuticals
- Atugen
- CBR Institute for Biomedical Research
- CytRx
- Duke University
- Exegenics
- Eyetech
- Froptix
- Harvard Medical School
- Inex Pharmaceuticals
- Intradigm
- Invitrogen
- Isis
- Medtronic
- Merck
- MIT
- Nastech Pharmaceutical
- Novartis Ophthalmics
- Opko Health
- OSI
- Pfizer
- Protiva Biotherapeutics
- RXi Pharmaceuticals
- Silence Therapeutics
- Sirna Therapeutics
- Tekmira Pharmaceuticals
- University of Massachusetts Medical School
| Format | Properties | |
|---|---|---|
| Electronic (PDF) | The report will be emailed to you. The report is sent in PDF format. | This is a single user license, allowing one specific user access to the product. |
| Enterprisewide | The report will be emailed to you. The report is sent in PDF format. | This is an enterprise license, allowing all employees within your organisation access to the product. |