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市場調查報告書
RNA治療市場動向
RNA Therapy: the next big thing after monoclonal antibodies?
| 出版商 |
Datamonitor |
| 出版日期 |
2008年10月 |
商品編碼 |
76542 |
| 內容資訊 |
英文 80 pages |
| 價格 |
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本報告已在2011年07月19日停止出版。
RNA治療雖然已研發多年，但還是得不到商業的成功。
本報告書內容包括：RNA治療研發生產線、技術、標的市場調查分析等，內容綱要摘記如下：
第1章 摘要
- 主要調查結果
- 為何要RNA治療
- 還沒證明正當性的RNA治療
- 長期銷售促進要素
- 高潛力吸引投資
- RNA治療能實現保證嗎
第2章 RNA治療背景
- 主要調查結果
- RNA治療受關注的理由
- RNA治療的生物學中心教條
- 把目標放在無法成為藥物研發標的的標的
- 歷史與發現
第3章 RNA治療與生產線概要
- 主要調查結果
- RNA治療誘因
- 各誘因RNA治療生產線分析
- Antisense oligonucleotide的最終階段生產線
- Antisense與siRNA集中，但變異型態也出現了
第4章 詳細產品與生產線
- 主要調查結果
- 各產品生產線
- 各企業生產線
- 生產中止產品
- RNA治療的銷售觀察
第5章 傳遞與治療領域分析
第6章 附錄
Abstract
Introduction
RNA therapies have been in development for a number of years, but are yet to
achieve any notable commercial success. Improvements have been made following
initial attempts and the technology may finally be coming to fruition.
Scope of this report
- Strategic review of the different RNA therapy technology offerings
- Assessment of the commercial potential of key products
- Analysis of the RNA therapy pipeline according to key metrics
- Guidance on the factors that dictate RNA therapy design and the markets
best suited to offer returns
Research and analysis highlights
With the loss of patent protection on maturing blockbusters, innovative new
products are required if the pharmaceutical industry is to maintain sales. RNA
therapy potentially provides a rich source of such products, allowing targets
that are not ' druggable' by other technologies to be exploited.
Despite a number of attempts, only one RNA therapy has reached the market.
However, new mechanisms such as RNA interference have given rise to a
generation of pipeline candidates characterized by improved safety and
efficacy.
While sales on the scale of those enjoyed by monoclonal antibodies seem a way
off, evidence suggests RNA therapy is now on course to deliver commercial
returns.
Key reasons to read this report
- Assess the 134 RNA therapies in preclinical development and beyond
- Understand how RNA therapies fit alongside small molecule drugs,
therapeutic proteins and monoclonal antibodies
- Understand reasons for the lack of approvals to date and how to maximize
chances of commercial success
Table of Contents
CHAPTER 1 EXECUTIVE SUMMARY
- Key findings
- Why RNA therapy?
- RNA therapy is not yet validated
- Longer-term sales driver
- High potential brings investment
- Can RNA therapy deliver on its promise?
CHAPTER 2 BACKGROUND TO RNA THERAPY
- Key findings
- Why the interest in RNA therapy?
- RNA therapies hit the central dogma of biology
- Gaining access to non-druggable targets
- mRNA splicing yields further potential targets
- Gene therapy also offers significant potential, but has fallen out of
favour
- History/ discovery
- Antisense silencing
- Antisense technology has been available for over 30 years
- Phenomenon of sense RNA also causing inhibition
- Emergence of RNA interference
- RNA interference discovered in 1998
- Accelerated entry of RNAi-based products into the clinic
CHAPTER 3 RNA THERAPY & PIPELINE OVERVIEW
- Key findings
- RNA therapy triggers
- Only one RNA therapy has reached the market to date
- Mechanisms of gene silencing
- RISC-independent silencing
- RISC-mediated (RNAi) pathway offers greater potency
- Analysis of the RNA therapy pipeline by trigger
- Late-stage pipeline largely consists of antisense oligonucleotides
- Antisense RNA therapies are at a more advanced stage of development
- Focus has shifted towards RISC-mediated silencing (RNAi) and siRNA
- Next-generation structures maintain interest in antisense
- Focus on antisense and siRNA, but variants are emerging
- Pipeline is dominated by siRNA and antisense
- Further silencing technologies are emerging
- miRNA offers a wealth of additional targets
CHAPTER 4 DETAILED PRODUCT PIPELINE
- Key findings
- Pipeline by product
- Late-stage pipeline
- Vitravene (formivirsen): only marketed RNA therapy
- Genasense (oblimersen): struggle to demonstrate efficacy
- Mipomersen (ISIS 301012): setbacks, but strong profile
- Alicaforsen: Phase III failure for Crohn' s disease
- Bevasiranib: challenging Lucentis in the maintenance of AMD
- Early-stage clinical pipeline
- Pre-clinical pipeline
- Pipeline by company
- Discontinued products
- Forecast RNA therapy sales
- Comparison to the uptake of monoclonal antibodies
CHAPTER 5 DELIVERY AND THERAPY AREA ANALYSIS.
- Key findings
- Therapy area analysis
- Pipeline by therapy area
- Novel technology requires high levels of unmet need
- Therapeutic focus driven by delivery technology
- Therapy area by trigger type
- Delivery remains key to success
CHAPTER 6 APPENDIX
- References
- Journals
- Websites
- Datamonitor reports
- Abbreviations
List of Tables
- Table 1: RNA therapy pipeline, marketed to Phase III
- Table 2: RNA therapy pipeline, Phase II and Phase I
- Table 3: RNA therapy pipeline, pre-clinical
- Table 4: Discontinued RNA therapy products
- Table 5: Segmented RNA therapy sales to 2020, $m
List of Figures
- Figure 1: RNA therapy offers access to novel, disease-relevant targets
- Figure 2: RNA therapy pipeline by development stage
- Figure 3: Illustrative RNA therapy market to 2020, $m
- Figure 4: Market capitalization of leading RNA therapy companies
- Figure 5: Considerations for successful RNA therapy development
- Figure 6: The central dogma of biology and potential role of RNA therapy
in relation to traditional ' drugging' approaches
- Figure 7: Technological advances expand the ' druggable' space
- Figure 8: Key milestones in RNA therapy
- Figure 9: RNA therapy pipeline by development stage
- Figure 10: RNA therapy pipeline by involvement of the RNA-induced
silencing complex, RISC
- Figure 11: Classification of RISC-independent silencing triggers
- Figure 12: Cellular processes involved in gene silencing
- Figure 13: Classification of RISC-mediated (RNAi) gene silencing triggers
- Figure 14: RNA therapy pipeline by development stage, split by silencing
trigger technologyFigure 15: RNA therapy pipeline by silencing trigger
technology , split by development stage
- Figure 16: RNA therapy pipeline by development stage and company
- Figure 17: RNA therapy pipeline by company, split by development stage
(largest weighted companies only)
- Figure 18: RNA therapy pipeline by company, split by trigger type (largest
weighted companies only)
- Figure 19: Estimated RNA therapy market to 2020, $m
- Figure 20: Comparison of estimated RNA therapy sales versus initial growth
of the monoclonal antibody market, $m
- Figure 21: RNA therapy pipeline by development stage and therapy area
- Figure 22: RNA therapy pipeline by therapy area, split by trigger type
- Figure 23: Challenges associated with RNA therapy delivery
- Figure 24: Administration routes used for RNA therapy
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