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RNA therapy: the next big thing after monoclonal antibodies?

  • Publication Date:October 2008
  • Publisher:Datamonitor
  • Product Type: Report
  • Pages:80

RNA therapy: the next big thing after monoclonal antibodies?

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

  • 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

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.

Reasons to Purchase

  • 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
  • Chapter 1 Executive Summary
  • Key Findings
    • Why Rna Therapy?
    • Rna Therapy Is Not Yet Validated
    • LongerTerm 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 NonDruggable 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 RnaiBased 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
    • RiscIndependent Silencing
    • RiscMediated (Rnai) Pathway Offers Greater Potency
  • Analysis Of The Rna Therapy Pipeline By Trigger
    • LateStage Pipeline Largely Consists Of Antisense Oligonucleotides
    • Antisense Rna Therapies Are At A More Advanced Stage Of Development
    • Focus Has Shifted Towards RiscMediated Silencing (Rnai) And Sirna
    • NextGeneration 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
    • LateStage 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
    • EarlyStage Clinical Pipeline
    • PreClinical 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
    • Administration
  • 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, PreClinical
    • 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, DiseaseRelevant 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 RnaInduced Silencing Complex, Risc
    • Figure 11: Classification Of RiscIndependent Silencing Triggers
    • Figure 12: Cellular Processes Involved In Gene Silencing
    • Figure 13: Classification Of RiscMediated (Rnai) Gene Silencing Triggers
    • Figure 14: Rna Therapy Pipeline By Development Stage, Split By Silencing Trigger Technology
    • Figure 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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