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市場調查報告書
RNAi治療：RNAi給藥的新策略
Realizing the Promise of Therapeutic RNAi: Emerging Strategies for RNAi Delivery
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RNAi治療：RNAi給藥的新策略 是由出版商Decision Resources, Inc.在2008年01月所出版的。
這份英文市場調查報告書包含26 Pages 價格從美金2250起跳。
本報告書內容包括：RNAi治療藥的給藥市場調查、RNA的機制、治療藥設計、各企業的RNAi治療藥給藥方式分析、主要企業介紹、未來市場預測等。內容綱要摘記如下：
介紹
RNA干涉的機制
RNAi研究測試藥及治療藥設計
RNAi治療藥的給藥方式
- Alnylam的未修飾siRNA直接給藥
- Nastech Pharmaceutical的載體運輸技術
- 利用SNALP的siRNA組織性給藥
- Alnylam Pharmaceuticals/MIT的脂質奈米粒子
- Alnylam的臨床流程
- Silence Therapeutics的siRNA-lipoplexes(脂質複合體)
- RXi的干涉奈米粒子
- Intradigm的TargeTran™奈米粒子
- 以分子為標的的siRNA的Covalent Conjugation
- siRNA抗體媒介的標的
- siRNA中樞神經的組織性標的
企業介紹
- Alnylam Pharmaceuticals
- Sirna Therapeutics
- RXi Pharmaceuticals
- Silence Therapeutics
RNAi治療藥給藥技術預測
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
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