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

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
- 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, 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