DNA/RNA療法：ゲノムがもたらす12億米ドル市場

Introduction

The DNA/RNA market is in its infancy with no significant products launched across the seven major markets. Despite the unproven nature of the sector in terms of market potential, it is commanding considerable interest across the biotechnology industry. Datamonitor has identified 99 companies developing DNA/RNA therapies, with 229 products in development.

Scope of this report

• Benchmarking of the current and future DNA/RNA market, by technology and therapeutic focus, in terms of number of projects and dollar value
• Analysis of market trends and future opportunities for both biotech and pharmaceutical players
• Investigation of the steps that the industrys emerging players are taking to increase innovation in their businesses and improve strategic position
• Full profiles of 19 DNA/RNA programs set to launch by 2010, encompassing product development and company strategy

Research and analysis highlights

The DNA/RNA market will not receive significant attention from the wider pharmaceutical industry until a series of antisense, aptamer and gene therapies are launched and achieves significant uptake across the seven major markets. The first product to achieve this is likely to be Eyetech/Pfizers Macugen.

Oncology dominates the DNA/RNA market, both in terms of commercial potential and pipeline development, while antisense and gene therapies lead the technological focus of those products set to launch by 2010.

Key reasons to read this report

• Forecast how advances in science and changes in the commercial environment will offer new opportunities to benefit from DNA/RNA therapies
• Identify key pipeline products for in-licensing opportunities as well as areas of unmet need
• Benchmark your position within the DNA/RNA market and identify the optimum strategy for future success

CHAPTER 1 EXECUTIVE SUMMARY

Scope

Datamonitors insight into the DNA/RNA market in 2004

The DNA/RNA market is forecast to generate $1.2bn by 2010, using a risk-adjusted market forecast, of which 45% is set to be generated by Pfizer/Eyetechs ophthalmic aptamer Macugen (pegaptanib). This product is set to drive the overall growth of the market, and it represents the optimal business development and innovation profile.

Macugens strong revenue-generating potential means that the ophthalmology franchise and the aptamer platform will dominate the focus of the DNA/RNA market, in terms of sales. Behind ophthalmology, oncology will dominate the therapeutic focus of the DNA/RNA market, and is set to generate 31.2% of 2010 sales.

After aptamers, antisense and gene therapies represent the leading technological focus of products set to launch by 2010 in terms of sales, together generating 46% of 2010 sales.

A critical step for emerging DNA/RNA therapeutics players before reaching full integraration will be to show they are capable of advancing with confidence a number of products beyond Phase III. Vital to this process will be their pipelines validation through strategic alliances with leading biopharma players.

CHAPTER 2 BUSINESS MODEL DYNAMICS

• Introduction
• Where is the DNA/RNA market currently?
• DNA/RNA market evolution
• DNA/RNA company and market analysis
• Drivers and resistors to DNA/RNA market evolution: a PESTEL analysis
• Political, Ethical and Social issues
• Will gene therapy be limited to severe, life-threatening indications with a high unmet need?
• Is it right to delay future gene therapy trials while we wait for safer vectors?
• Are there any issues that are relevant for ex-vivo gene therapy versus in-vivo gene therapy?
• Will gene therapy be limited to somatic gene therapy, rather than germline gene therapy?
• Conclusion
• Recommendation
• Economics: Pharmacoeconomic issues
• Acute vs. chronic treatments
• Drug development
• Physician mentality
• Company business plan flexibility
• Pricing and logistical issues
• Recommendation
• Legal: Intellectual Property and Regulatory Approval
• Gene therapy
• Antisense
• RNAi, aptamers, ribozymes and DNA
• Most active DNA/RNA companies
• Conclusion
• Regulatory issues
• Recommendation
• DNA/RNA market and company strategy
• Alliance strategies - licensing and R&D collaboration
• Originator collaborations
• Development/commercialization collaborations
• Drivers and resistors of collaborations
• FIPCO/FIBio collaborations
• Collaborations with earlier-stage companies
• Mergers and acquisitions
• Product development strategy
• Winning strategies

CHAPTER 3 TECHNOLOGICAL AND THERAPEUTIC EVOLUTION

• Classifying the DNA/RNA market
• Gene therapy
• Evolution of vector development
• DNA viral vectors
• RNA viral vectors
• Non-viral vectors
• Market dynamics of vector choice
• Current state of the gene therapy market
• Future gene therapy market dynamics
• Market leaders
• Antisense
• Evolution
• First-generation antisense and immunomodulatory antisense
• Second-generation antisense
• Further evolution of antisense technology
• Antisense market dynamics
• Current state of the antisense market
• Future antisense market dynamics
• Market leaders
• RNAi, aptamers, ribozymes and DNA
• Categories
• RNAi
• Aptamers
• Ribozymes
• DNA
• RNAi, aptamers, ribozymes and DNA market dynamics
• Current state of the antisense market
• Future RNAi, aptamer, ribozyme and DNA market dynamics
• Market leaders
• Delivery modality
• 1. Vectors
• 2. Direct delivery
• Delivery modality profiles of products set to launch by 2010
• Innovative delivery modalities in development
• RASONs
• Gene pill
• Pathway IV
• Therapeutic diversification
• Key therapeutic development trends across the DNA/RNA market
• Technological diversification
• Key technological development trends across the DNA/RNA market
• Productivity leaders
• Technological focus of DNA/RNA productivity leaders
• Therapeutic focus of DNA/RNA market leaders

CHAPTER 4 PORTFOLIO AND PIPELINE ANALYSIS

• Active DNA/RNA programs overview, 2004
• Methodology of analysing and forecasting products set to launch by 2010
• 1. First-year sales and impacting sales and marketing collaborations
• 2. Absolute and risk-adjusted market forecasting
• 3. Sourcing information
• DNA/RNA market, 2004
• The DNA/RNA market to 2010
• Total and risk adjusted DNA/RNA market forecast to 2010
• Gene therapies
• Products scheduled to launch by 2010
• Gendicine
• Innovation
• Business development
• DS-992
• Innovation
• Business development
• Advexin (INGN-201)
• Innovation
• Business development
• ANG1 (VEGF gene therapy)
• Innovation
• Business development
• Cerepro (EG009)
• Innovation
• Business development
• BioBypass (AdGVVEGF121.10)
• Innovation
• Business development
• tgAAVCF
• Innovation
• Business development
• Gene therapy products not scheduled to launch by 2010
• Antisense therapies
• Products scheduled to launch by 2010
• E2F decoy (edifoligide, CGT-003, E2F-D)
• Innovation
• Business development
• Genasense (oblimersen sodium)
• Innovation
• Business development
• AP 12009
• Innovation
• Business development
• Resten-NG (AVI-4126)
• Innovation
• Business development
• ProMune (CPG 7909)
• Innovation
• Business development
• Kappaproct
• Innovation
• Business development
• Vitravene (fomivirsen)
• Innovation
• Business development
• ISIS14803
• Innovation
• Business development
• Alicaforsen (ISIS2302)
• Innovation
• Business development
• ISIS104838
• Innovation
• Business development
• Antisense therapy products not scheduled to launch by 2010
• RNAi, aptamers, ribozymes and DNA
• Products scheduled to launch by 2010
• Macugen (pegaptanib)
• Innovation
• Business development
• Ampligen (polyI:polyC12U)
• Innovation
• Business development
• RNAi, aptamer, ribozyme and DNA products not scheduled to launch by 2010

CHAPTER 5 APPENDIX : SUPPORTING DATA

• Financial data
• Exchange rates
• Methodology for therapy area forecasting

List of Tables

• Table 1: Patenting activity of most active DNA/RNA companies
• Table 2: Drivers and resistors of collaborations in the DNA/RNA market
• Table 3: Key deals of most productive DNA/RNA companies, 1996-2003
• Table 4: Winning strategies in the DNA/RNA market
• Table 5: Main characteristics of commonly investigated gene therapy vector types
• Table 6: Main characteristics of commonly investigated antisense platform types
• Table 7: DNA/RNA market, 2004
• Table 8: Market potential of each product set to launch by 2010
• Table 9: Scoring of each product forecast to be launched by 2010, to create a percentage probability of successful launch
• Table 10: Exchange rates, 2003

List of Figures

• Figure 1: The structure of Datamonitors analysis of the DNA/RNA market, 2004
• Figure 2: Risk-adjusted total DNA/RNA market forecast, to 2010
• Figure 3: Total number of active DNA/RNA programs, split by therapeutic focus
• Figure 4: Total number of active DNA/RNA programs, split by technological focus
• Figure 5: Balance between business development and innovation for products set to be launched between 2005-2010
• Figure 6: Technology focus for active gene therapy programs, 2004
• Figure 7: New gene therapy trials approved by year, 1989-2003
• Figure 8: Vector focus of most active gene therapy players, 2004
• Figure 9: Pipeline development focus of antisense subclasses, 2004
• Figure 10: Platform focus of most productive antisense players, 2004
• Figure 11: Pipeline development focus of RNAi, aptamers, ribozymes and DNA products, 2004
• Figure 12: Technology focus of most active RNAi, aptamers, ribozymes and DNA players, 2004
• Figure 13: Commercial and developmental therapeutic focus of the DNA/RNA market, 2004
• Figure 14: Commercial and developmental technological focus of the DNA/RNA market, 2004
• Figure 15: Technological focus of most active DNA/RNA players, 2004
• Figure 16: Therapeutic focus of most active DNA/RNA players, 2004
• Figure 17: Timeline of forecast DNA/RNA product launches to 2010
• Figure 18: Unadjusted total DNA/RNA market forecast, to 2010
• Figure 19: Risk-adjusted total DNA/RNA market forecast, to 2010
• Figure 20: Biotech sector sales growth, 2003-2008
• Figure 21: Gene therapy programs set to launch by 2010, split by technology focus and therapy
• Figure 22: Early clinical gene therapy programs, split by therapy and technology focus
• Figure 23: Preclinical gene therapy programs, split by therapy and technology focus
• Figure 24: Antisense therapy programs set to launch by 2010, split by technology focus and therapy
• Figure 25: Early clinical antisense therapy programs, split by therapy and technology focus
• Figure 26: Preclinical antisense programs, split by therapy and technology focus
• Figure 27: Early clinical RNAi, aptamers, ribozymes and DNA therapy programs, split by therapy and technology focus
• Figure 28: Preclinical RNAi, aptamers, ribozymes and DNA therapy programs, split by therapy and technology focus