RNAi治療藥物：第二代候選藥物之契機 是由出版商Insight Pharma Reports在2010年10月所出版的。
這份英文市場調查報告書包含136 Pages 價格從美金3195起跳。
本報告為，分析RNAi治療藥物之開發趨勢，並匯整RNA干擾設計、傳遞系統、市場參與主要企業簡介、大型製藥公司角色、今後展望及專家意見等，以下列摘要形式闡述。
實施概要
第1章 簡介
第2章 小沈默RNA、RNA干擾機轉
- RNAi經由
- siRNA及miRNA之轉錄後基因沉默
第3章 RNA干擾設計
- 臨床開發中之RNAi治療藥物
- 寡核苷酸用於治療RNAi藥劑之課題
- 避免本質性免疫活性化所需之siRNA之設計
- 主要企業開發之替代干擾寡核苷酸設計
- 結論
第4章 RNAi給藥媒介開發
- siRNA治療藥物之直接給藥
- siRNA全身給藥所需Tekmira之SNALP技術利用
- RXi之siRNA全身給藥平台
- Calando之Rondel傳遞系統，其他
第5章 治療用RNAi領域主要企業
- Alnylam Pharmaceuticals
- Tekmira Pharmaceuticals
- Sirna Therapeutics/Merck
- RXi Pharmaceuticals
- Calando Pharmaceuticals
- Silence Therapeutics
- Quark Pharmaceuticals
- Dicerna Pharmaceuticals
- Summary table
- siRNA治療藥物開發中大型製藥公司之角色
第6章 miRNA治療藥物之主要企業
- Regulus Therapeutics
- Santaris Pharma
- miRagen Therapeutics
- Summary table
- miRNA治療藥物開發中大型製藥公司之角色
第7章 RNAi治療藥物之今後展望
第8章 專家訪談
參考資料
企業名錄（附網址）
Abstract
Many established pharma companies have entered into major deals with some of
these RNAi therapeutics companies. Also covered are companies focus on
development of microRNA-based therapeutics and diagnostics. These have
attracted the interest of the investment community and the pharmaceutical
industry. This report discusses the outlook for the therapeutic RNAi/miRNA
industry sector, including strategic issues such as technological prematurity
and the development of enabling technologies, the role of Big Pharma
investment, the impact of patent litigation and cross-licensing in shaping the
RNAi/miRNA sector, and a scenario for the development of drugs in the
therapeutic RNAi/miRNA sector.
Key topics covered include:
- Delivery vehicle technologies
- Oligo protective chemistries
- Therapeutic indications in development
- Phase I and II development highlights
- Big Pharmas' investments, activities and strategies
- Profiles of leading companies
Following an overview of the discovery and evolution of the RNAi and miRNA
field, RNAi Therapeutics: Second-Generation Candidates Build Momentum turns to
the science behind therapeutic RNAi and miRNA, technologies for design of
therapeutic oligonucleotides that work via an RNAi or miRNA-modulating
mechanism, technologies for design of delivery vehicles, and leading companies
in the therapeutic RNAi/miRNA industry sector as well as the role of large
pharmaceutical companies in the sector.
Selected leading therapeutic RNAi specialty companies discussed in Chapter 5
"RNA interference" (RNAi) refers to the inhibition of expression of specific
genes by small doublestranded RNAs. Since the emergence of RNAi in the late
1990s, it has been seen in the medical community as breakthrough science. More
recently, the biology of microRNAs (miRNAs), a class of naturally occurring
small RNAs, has also been found to be involved in gene regulation. The gene
regulatory function of these molecules has generated intense interest in the
development of oligonucleotide drugs to address targets that are considered to
be "undruggable" by small-molecule compounds or biologics. The history of the
development of antisense and aptamer drugs, though difficult, is providing the
foundation for the successful development of oligonuclotide drugs. This has
resulted in developing chemical modifications to prevent their destruction by
nucleases, improve their pharmacokinetics, and enhance their target
specificity and binding affinity.
RNAi Therapeutics: Second-Generation Candidates Build Momentum includes a
review of RNAi company pipelines as well as activities within large
pharmaceutical companies. Many established pharma companies have entered into
major deals with some of these RNAi therapeutics companies. Also covered are
companies' focus on development of microRNA-based therapeutics and
diagnostics. These have attracted the interest of the investment community and
the pharmaceutical industry. This report discusses the outlook for the
therapeutic RNAi/miRNA industry sector, including: strategic issues such as
technological prematurity and the development of enabling technologies, the
role of Big Pharma investment, the impact of patent litigation and
cross-licensing in shaping the RNAi/miRNA sector, and a scenario for the
development of drugs in the therapeutic RNAi/miRNA sector.
Table of Contents
EXECUTIVE SUMMARY
Chapter 1 INTRODUCTION
- 1.1 Development of Other Types of Oligonucleotide Therapeutics has been
Leading the way to Developing RNAi Therapeutics
- 1.2 Implications of the Development of Antisense and Aptamer Drugs for the
Therapeutic RNAi Field
Chapter 2 SMALL SILENCING RNAs, AND MECHANISMS OF RNA INTERFERENCE
- 2.1 The RNAi Pathway
- microRNAs: Processing and Silencing Pathways
- PIWI-interacting RNAs
- 2.2 Transcriptional Gene Silencing by siRNA and miRNA
- Small Silencing RNAs in the Universe of Small Regulatory RNAs
Chapter 3 DESIGN OF INTERFERING RNAs
- 3.1 RNAi Therapeutics Now in Clinical Development
- 3.2 Challenges in Designing Oligonucleotides as Therapeutic RNAi Agents
- 3.3 Design of siRNAs to Avoid Innate Immune Activation
- siRNA therapeutics designed to trigger both immunostimulation and
specific RNAi
- Getting naked RNAs into the cell in experimental in vitro and in vivo
studies of potential RNA therapeutics
- 3.4 Alternative Designs of Interfering Oligonucleotides Being Developed by
Leading Companies
- Dicerna' s Dicer Substrate Technology
- RXi' s proprietary RNAi compounds
- rxRNAori
- rxRNA solo
- sd-rxRNA
- 3.5 Conclusions
Chapter 4 DEVELOPMENT OF DELIVERY VEHICLES FOR RNAi DRUGS
- 4.1 Direct Delivery of siRNA Therapeutics
- Alnylam/Kyowa Hakko Kirin/ Cubist' s ALN-RSV01
- Pfizer/Quark/Silence' s PF-4523655
- RXi' s sd-rxRNA platform for local delivery
- 4.2 Alnylam' s Use of Tekmira' s SNALP Technology for System Delivery of
siRNA Therapeutics to the Liver
- Second-generation SNALP technologies
- 4.3 RXi' s siRNA Systemic Delivery Platforms
- 4.4 Calando' s Rondel Delivery System
- 4.5 Silence Therapeutics' s AtuPLEX and PolyTran Delivery Platforms
- AtuPLEX delivery system
- PolyTran delivery system
- 4.6 Delivery Vehicle for siRNA Designed to Cross the Blood-Brain Barrier
- 4.7 Conclusions
Chapter 5 LEADING COMPANIES IN THE THERAPEUTIC RNAi FIELD
- 5.1 Alnylam Pharmaceuticals
- Alnylam' s strategy
- Alnylam' s collaborations
- Alnylam' s candidate products
- Alnylam' s ventures
- 5.2 Tekmira Pharmaceuticals
- 5.3 Sirna Therapeutics/Merck
- 5.4 RXi Pharmaceuticals
- 5.5 Calando Pharmaceuticals
- 5.6 Silence Therapeutics
- 5.7 Quark Pharmaceuticals
- QPI-1002
- QPI-1007
- Quark' s collaborations
- Quark' s 2010 $10 million financing
- 5.8 Dicerna Pharmaceuticals
- Dicerna' s collaborations
- 5.9 Summary table
- 5.10 The Role of Big Pharma in the Development of siRNA Therapeutics
Chapter 6 LEADING COMPANIES IN miRNA THERAPEUTICS
- 6.1 Regulus Therapeutics
- Regulus' alliances
- Regulus' products
- Regulus' "tool kit" for designing miR-122 antagonists and antagonists to
other miRNAs
- miR-21 and fibrosis
- Other miRNA-based discovery/development programs at Regulus
- 6.2 Santaris Pharma
- Santaris' partners
- Santaris' pipeline
- 6.3 miRagen Therapeutics
- miRagen' s technology platform
- miRagen' s alliances
- miRagen' s pipeline
- 6.4 Summary table
- 6.5 The Role of Big Pharma in the Development of miRNA Therapeutics
Chapter 7 OUTLOOK FOR RNAi THERAPEUTICS
- 7.1 Is Therapeutic RNAi Technologically Premature?
- 7.2 Why is Big Pharma so Interested in Investing in RNAi and miRNA
Therapeutics?
- 7.3 Patents, Lawsuits and the Development of the RNAi/miRNA Sector
- 7.4 A Scenario for the Development of Drugs in the Therapeutic RNAi/miRNA
Sector
Chapter 8 EXPERT INTERVIEWS
- 8.1. Dalia Cohen, PhD, Chief Scientific Officer, Rosetta Genomics,
Philadelphia, PA
- 8.2. Roberto Guerciolini, MD, Senior Vice President, Pharmaceutical
Developmen, Dicerna Pharmaceuticals, Watertown, MA
- 8.3. Arthur M. Krieg, MD, Chief Scientific Officer, Oligonucleotide
Therapeutics Unit, Pfizer, Cambridge, MA
- 8.4. William S. Marshall, PhD,President and Chief Executive Officer,
miRagen Therapeutics, Boulder, CO
- 8.5. James D. Thompson, PhD, Vice President, Pharmaceutical Development,
Quark Pharmaceuticals, Boulder, CO
REFERENCES
COMPANY INDEX WITH WEB ADDRESSES
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