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Biotechnology

Ribonucleic Acid (RNA) Therapeutics

Strong Potential to Address Unmet Needs in Pharma

Publication Date February 2010
Publisher GBI Research
Product Type Report
Pages 89
ISBN Number not applicable
Product Code GBI00026

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£2,310.00
approximately: $3,498 | €2,578

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Summary

GBI Research, the leading business intelligence provider, has released its latest research "Ribonucleic Acid (RNA) Therapeutics - Strong Potential to Address Unmet Needs in Pharma", which provides insights into the unmet needs in the pharmaceutical and healthcare market and potential segments where RNA therapies can prove to be useful. This pharma report also delves deep into the market potential that RNA therapies hold in the future. Further, it provides insights into the R&D pipeline and the promising drugs. The study elucidates the current competitive landscape of the RNA therapeutics market. Finally, the report deals with key trend analysis on mergers and acquisitions, and licensing agreements involving RNA therapies.

This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research's team of industry experts.

Scope

The scope of this report includes:

  • Detailed market characterization including evaluation of market potential, annual cost of therapy and treatment usage patterns
  • Key technologies that have created significant impact on research and development of RNA therapeutics
  • Key factors that can drive the RNA therapeutics market potential
  • Key risks and issues associated with various RNA therapies
  • Competitive landscape of RNA therapeutics market. The key companies studied in this report are Antisense Pharma GmbH, Lorus Therapeutics, Isis Pharmaceuticals, Alfacell Corporation, Genta Incorporated, Atlantic Healthcare, Alnylam, OPKO, Oxford BioMedica, Dicerna Pharmaceuticals, Silence Therapeutics plc, Cequent Pharmaceuticals, Inc and Santaris Pharma.
  • Key M&A activities and licensing agreements that have taken place between 2008 and 2009 in the RNA therapeutics market.

Reasons to buy

The report will aid business development and marketing executives in strategizing their product launches and help venture capitalists to identify promising small and medium sized enterprises. It will allow you to

  • Build effective strategies to launch pipeline products by identifying potential geographies
  • Exploit in-licensing and out-licensing opportunities by identifying products that will fill the portfolio gaps
  • Develop key strategic initiatives by studying the key strategies of top competitors
  • Identify potential SME companies that hold a lot of promise
  • Reinforce R&D pipelines by identifying new target mechanisms which can produce first in class molecules with more efficiency and better safety

Contents

  • 1 Table of Contents
    • 1.1 List of Tables
    • 1.2 List of Figures
  • 2 RNA Therapeutics: Introduction
    • 2.1 GBI Research Report Guidance
  • 3 RNA Therapeutics - Introduction to Ribonucleic Acid (RNA)
    • 3.1 What is RNA Therapy?
      • 3.1.1 RNA is the Key for Protein Synthesis
    • 3.2 Blocking Translation of mRNA Inhibits Protein Biosynthesis, the Key for Cell Proliferation
    • 3.3 Role of RNA in Pharmaceutical Industry
      • 3.3.1 Upstream Action of RNA Therapies Make them more effective Than the Existing Biologic Therapies
  • 4 RNA Therapeutics - Overview on RNA Technology
    • 4.1 Antisense RNA
      • 4.1.1 Therapeutic Objective
      • 4.1.2 Issues with Antisense RNA Therapy
      • 4.1.3 Solutions Developed to Overcome Issues
    • 4.2 RNA Interference Technology
      • 4.2.1 siRNA
      • 4.2.2 Micro RNA
      • 4.2.3 Issues with Interfering RNA Therapy
      • 4.2.4 Soultions Developed to Overcome Issues
    • 4.3 RNA Therapies Are Expected To Create Significant Impact on the Current Therapies After They Enter the Market
      • 4.3.1 Current Therapies in the Market Are Expected to Witness Strong Competition from RNA Therapies Once They Enter the Market
      • 4.3.2 RNA Therapies Have Created a Significant Impact on Major Funding Institutions Such As Venture Capitalist Firms and Private Equity Firms
      • 4.3.3 The Impact of RNA Therapies on Patients Is Expected To Grow Gradually Since Patients Would Switch Over Only After RNA Therapies Pass through Proof-Of-Concept Studies
      • 4.3.4 RNA Therapeutics Has Already Made a Significant Impact on Large Pharmaceutical and Biotech Companies
    • 4.4 Key Challenges Such As Lack of Effective Delivery Systems are Holding Back RNA Therapies from Entering the Market
  • 5 RNA Therapeutics - Market Characterization
    • 5.1 Market Potential
    • 5.2 Target Sales Volume
    • 5.3 Annual Cost of Therapy
    • 5.4 Technology Adoption Framework
      • 5.4.1 RNA Technology has the potential to meet Unmet-needs in Major Diseases
      • 5.4.2 RNA technology Failed in Proof-of-concept Studies
      • 5.4.3 Lack of awareness among patients as well as physicians is embracing the adoption of RNA Technology
      • 5.4.4 RNA therapies, launched till now, failed to pass through proof-of-concept studies
    • 5.5 RNA Therapeutics Market Drivers
      • 5.5.1 Dual Mechanism of Action is the Major Factor Driving RNA Therapeutics Market Potential
      • 5.5.2 Failure of Conventional Therapies in Curing Chronic Diseases has Created High-unmet Needs
      • 5.5.3 Major Break-through Technologies in RNA Research and Development Opened-up New Markets
      • 5.5.4 Increased Funding on RNA Research by Major Countries such as the US and Europe
    • 5.6 RNA Therapeutics Market Restraints
      • 5.6.1 Many RNA Therapies are Failing at Phase III Clinical Development
      • 5.6.2 The Characteristics of RNA Structure make them Vulnerable to Human Enzymes
      • 5.6.3 Lack of Effective Delivery Technologies That can Deliver RNA therapies to the Target Site are making RNA Therapies Ineffective
      • 5.6.4 RNA therapies can cause potential side-effects and off-target effects
  • 6 RNA Therapeutics - Market Description
    • 6.1 SWOT Analysis
      • 6.1.1 RNA Therapies Hold a Competitive Edge over Conventional Therapies Due to Their Gene Silencing Ability
      • 6.1.2 Although RNA Therapies Have the Potential to Cure Diseases, Few Drawbacks Such As Instability and Off-targets are Holding Them from Displaying Desired Efficacy Levels
      • 6.1.3 Abundant Opportunities in Terms of Unmet Needs Exist for RNA Therapies in Chronic disease Conditions Associated with Genetic Mutations and Protein Overexpression
      • 6.1.4 Similar to RNA Therapies, Other Treatments such as Stem Cell Therapies Are Also in Research and Development, and These Therapies Can Pose a Threat to RNA Therapeutics Market Potential
    • 6.2 Porters Five Forces Analysis on RNA Therapeutics
      • 6.2.1 Threat of Substitutes
      • 6.2.2 Threat of Potential Entrants
      • 6.2.3 Bargaining Power of Suppliers
      • 6.2.4 Bargaining Power of Buyers
    • 6.3 PEST Analysis
      • 6.3.1 The US
      • 6.3.2 The Top five Countries in Europe
      • 6.3.3 Japan
    • 6.4 Funding
      • 6.4.1 US
      • 6.4.2 Europe
  • 7 RNA Therapeutics - Competitive Landscape
    • 7.1 Key Players
      • 7.1.1 Antisense Pharma GmbH
      • 7.1.2 Lorus Therapeutics
      • 7.1.3 Isis Pharmaceuticals
      • 7.1.4 Alfacell Corporation
      • 7.1.5 Genta Incorporated
      • 7.1.6 Atlantic Healthcare
      • 7.1.7 Alnylam
      • 7.1.8 OPKO
    • 7.2 Promising Companies Developing RNAi under Collaborations with major Companies
      • 7.2.1 Oxford BioMedica
      • 7.2.2 Dicerna Pharmaceuticals
      • 7.2.3 Silence Therapeutics plc
      • 7.2.4 Cequent Pharmaceuticals, Inc
      • 7.2.5 SantarisPharma A/S
  • 8 RNA Therapeutics - R&D Pipeline Analysis
    • 8.1 Strategic Pipeline Assessment
    • 8.2 Profiles of Key Late-Stage Drugs
      • 8.2.1 Mipomersen
      • 8.2.2 Bevasiranib
      • 8.2.3 LOR-2040
      • 8.2.4 Genasense
      • 8.2.5 Trovax
      • 8.2.6 Trabedersen
      • 8.2.7 Onconase
      • 8.2.8 Alicaforsen
    • 8.3 Insights into Most Promising Drugs
      • 8.3.1 Mipomersen
      • 8.3.2 LOR-2040
      • 8.3.3 Genasense
      • 8.3.4 ProSavin
      • 8.3.5 Trovax
      • 8.3.6 Trabedersen
      • 8.3.7 Onconase
      • 8.3.8 Alicaforsen
  • 9 RNA Therapeutics - M&A Landscape
    • 9.1 Big Pharmaceutical Companies Relentlessly Attempting to Unlock RNA therapy Potential
    • 9.2 Influx of Huge Venture Capitalist Funding into Small and Medium Biotech Companies Venturing into RNA Therapeutics Development
      • 9.2.1 Dicerna Pharmaceuticals
      • 9.2.2 Cequent Pharmaceuticals
      • 9.2.3 Prosensa
      • 9.2.4 Cylene Pharmaceuticals
    • 9.3 High Value Collaborations involving RNA Therapies
      • 9.3.1 ISIS Pharmaceuticals
      • 9.3.2 Alnylam
      • 9.3.3 Silence Therapeutics and AstraZeneca
      • 9.3.4 Santaris Pharma and Wyeth Pharmaceuticals
    • 9.4 Large Pharmaceutical and Biotechnology companies are vying for major acquisitions in the RNA Therapeutics Market
      • 9.4.1 Thermo Fisher Scientific and Open Biosystems
      • 9.4.2 Roche and Mirus Bio
    • 9.5 R&D Licensing Agreements
      • 9.5.1 Deals by Phase
      • 9.5.2 Deals by Geography
      • 9.5.3 Deals by Licensing Type
      • 9.5.4 Deals by Value ($)
      • 9.5.5 Discovery/Development
      • 9.5.6 Preclinical
      • 9.5.7 Phase I
      • 9.5.8 Phase II
      • 9.5.9 Phase III
  • 10 RNA Therapeutics: Appendix
    • 10.1 Market Definitions
    • 10.2 Abbreviations
    • 10.3 Research Methodology
      • 10.3.1 Coverage
      • 10.3.2 Secondary Research
      • 10.3.3 Primary Research
      • 10.3.4 Forecasts
      • 10.3.5 Expert Panel Validation
    • 10.4 Contact Us
    • 10.5 Disclaimer
    • 10.6 Sources
  • 1.1 List of Tables
    • Table 1: RNA Therapeutics, Global, siRNA and shRNA Delivery Options, November 2009
    • Table 2: RNA Therapeutics, Global, Market Potential ($ m), 2005-2015
    • Table 3: RNA Therapeutics, Global, Diagnosis Population, 2011-2015
    • Table 4: RNA Therapeutics, Global, Antisense Pharma R&D Pipeline, November 2009
    • Table 5: RNA Therapeutics, Global, Lorus Therapeutics R&D Pipeline, November 2009
    • Table 6: RNA Therapeutics, Global, Isis Pharmaceuticals, R&D Pipeline, November 2009
    • Table 7: RNA Therapeutics, Global, Alfacell Corporation, R&D Pipeline, November 2009
    • Table 8: RNA Therapeutics, Global, Genta Inc., R&D Pipeline, November 2009
    • Table 9: RNA Therapeutics, Global, Alnylam, InterfeRx Licensing Agreements, November 2009
    • Table 10: RNA Therapeutics, Global, Alnylam, Licensing Agreements, November 2009
    • Table 11: RNA Therapeutics, Global, OPKO, Bevasirnanib Development History, November 2009
    • Table 12: RNA Therapeutics, Global, OPKO, Antisense Drugs Pipeline, November 2009
    • Table 13: RNA Therapeutics, Global, Oxford BioMedica, R&D Pipeline, November 2009
    • Table 14: RNA Therapeutics, Global, Silence Therapeutics, R&D Pipeline, November 2009
    • Table 15: RNA Therapeutics, Global, SantarisPharma, R&D Pipeline, November 2009
    • Table 16: RNA Therapeutics, Global, Discovery/Development Stage Drugs Licensing Agreements , 2008-2009
    • Table 17: RNA Therapeutics, Global, Preclinical Drugs Licensing Agreements , 2008-2009
    • Table 18: RNA Therapeutics, Global, Phase II Drugs Licensing Agreements, 2008-2009
    • Table 19: RNA Therapeutics, Global, Phase III Drugs Licensing Agreements, 2008-2009
    • Table 20: RNA Therapeutics, Global, Currency Conversion Rate, 2006-2008
  • 1.2 List of Figures
    • Figure 1: RNA Therapeutics, Global, Biosynthesis Process of Proteins
    • Figure 2: RNA Therapeutics, Global, Process of Blocking Synthesis of Disease-causing Proteins
    • Figure 3: RNA Therapeutics, Global, RNA Taxonomy
    • Figure 4: RNA Therapeutics, Global, Issues with Antisense RNA, November 2009
    • Figure 5: RNA Therapeutics, Global, Antisense RNA Drug Delivery Options, November 2009
    • Figure 6: RNA Therapeutics, Global, RNA interference Pathway
    • Figure 7: RNA Therapeutics, Global, Applications of RNAi-based Therapeutic Agents, November 2009
    • Figure 8: RNA Therapeutics, Global, miRNA Gene Silencing Pathway
    • Figure 9: RNA Therapeutics, Global, Issues with Interfering RNA, November 2009
    • Figure 10: RNA Therapeutics, Global, siRNA Key Drug Delivery Technologies, November 2009
    • Figure 11: RNA Therapeutics, Global, Impact Analysis, 2009
    • Figure 12: RNA Therapeutics, Global, Key R&D Steps Where RNA Therapies are Failing
    • Figure 13: RNA Therapeutics, Global, Market Potential ($ bn), 2005-2015
    • Figure 14: RNA Therapeutics, Global, Diagnosis Population (million), 2011-2015
    • Figure 15: RNA Therapeutics, Global, Indicative Pricing of RNA Therapies, 2009
    • Figure 16: RNA Therapeutics, Global, RNA Technology Adoption Framework
    • Figure 17: RNA Therapeutics, Global, Cost Vs Unmet Needs Comparison of Key Drug Classes
    • Figure 18: RNA Therapeutics, Global, Drivers and Restraints of Market Potential, 2009
    • Figure 19: RNA Therapeutics, Global, SWOT Analysis
    • Figure 20: RNA Therapeutics, Global, Porter's Five Forces Analysis
    • Figure 21: RNA Therapeutics, Global, US PEST Analysis
    • Figure 22: RNA Therapeutics, Global, Top Five Countries in Europe, PEST Analysis, 2009
    • Figure 23: RNA Therapeutics, Global, Japan, PEST Analysis, 2009
    • Figure 24: RNA Therapeutics, Global, US Medical Research Funding Framework, 2009
    • Figure 25: RNA Therapeutics, Global, Expected Competition from Exisiting Therapies
    • Figure 26: RNA Therapeutics, Global, RNA Therapies Benchmarking Against Monoclonal Antibodies
    • Figure 27: RNA Therapeutics, Global, R&D Pipeline Segmentation by Phase of Development, November 2009
    • Figure 28: RNA Therapeutics, Global, R&D Pipeline Segmentation by Therapeutic Class, November 2009
    • Figure 29: RNA Therapeutics, Global, R&D Pipeline Segmentation by Company, November 2009
    • Figure 30: RNA Therapeutics, Global, Preclinical Development Duration Comparison, November 2009
    • Figure 31: RNA Therapeutics, Global, Key M&A Activities Involving RNA Therapies, 2008-2009
    • Figure 32: RNA Therapeutics, Global, Major Licensing Agreements By Phase, 2008-2009
    • Figure 33: RNA Therapeutics, Global, Major Licensing Agreements By Geography, 2008-2009
    • Figure 34: RNA Therapeutics, Global, Major Licensing Agreements By Deal, 2008-2009
    • Figure 35: GBI Research Methodology
    • Figure 36: GBI Research Market Forecasting Model