Chapter 1 RNA Interference & Delivery
- Key Findings
- Introduction
- RNAi: a natural phenomenon
- Antisense technologies
- RNAi silencing agents
- RNAi silencing pathways
- microRNAs and antagomirs
- miRNA gene silencing
- Pros & Cons of RNAi silencing agents
- RNAi R&D
- High throughput in vitro screening
- RNAi in vivo screening
- RNAi diagnostic markers
- RNAi therapeutics
- RNAi diagnostics
- RNAi therapy – A Discovery Timeline
Major scientific challenges for the development of RNAi-based diagnostics & therapies
Chapter 2 RNAi-based Therapeutics: Pipeline Analysis
- Key points
- Key therapy areas covered by RNAi-based therapies
- Product pipelines
- RNAi for diagnostics
- RNAi companies analysed in this report
Chapter 3 RNAi-based Therapies for Cancer
- Key Findings
- Introduction
- RNAi delivery in cancer
- RNAi-based Therapies - siRNA
- Case study: ALN-VSP (Alnylam Pharmaceuticals)
- Case study: CALAA-01 (Calando Pharmaceuticals)
- Case study: Dicer substrate RNA (Dicerna Pharmaceuticals)
- Case study: Intratumoral siRNA (Genesis Research & Development Corporation)
- Case study: NPX delivery technology (Intradigm Corporation)
- Case study: SNS-01 (Senesco Technologies)
- Case study: Atu027 (Silence Therapeutics)
- Case study: STP702 (Sirnaomics)
- Case study: PLK SNALP (Tekmira Pharmaceutical Corporation)
- RNAi-based therapies - shRNA
- Case study: CEQ501 (Cequent pharmaceuticals)
- Alternative RNA approaches to develop cancer therapies
- miRNA in cancer
- Case study: miRNAs (Mirna Therapeutics)
- Case study: miRNAs (Regulus Therapeutics)
- Case study: SPC2996 & EZN3042 (Santaris Pharma A/S/Enzon Pharmaceuticals)
- MiRNA-based diagnostics
- mRNA antagonists
- Case study: LOR-2040 (Lorus Therapeutics)
- Antisense
- Case study: OGX-011 (Isis Pharmaceuticals/OncoGenex) & LY2181308 (Isis Pharmaceuticals/Eli Lilly)
- Case study: TF siRNA (siRNAsense A/S/Polyplus Transfection)
- Conclusions
- Our opinion on RNAi-based therapies & diagnostics for cancer
- Where the technology is now, its evolution, achievements and pitfalls
- Competition
- Potential future applications
- Activity in the market, major players and winners
Chapter 4 RNAi-based Therapies for CNS Disorders
- Key findings
- Introduction
- Neurodegenerative diseases
- Amyotrophic lateral sclerosis
- Case study: SOD1 siRNA (RXi Therapeutics)
- Case study: SOD1 shRNA (Oxford BioMedica)
- Huntington’s Disease
- Case study: ALN-HTT (Alnylam Pharmaceuticals/Medtronic)
- Case study: AVV-HTT (Targeted Genetics Corporation)
- Parkinson’s Disease
- Case study: SNCA-siRNA (Alnylam Pharmaceuticals)
- Case study: biotinylated siRNA (ArmaGen Technologies)
- Other CNS conditions
- Conclusions
- Our opinion on RNAi-based therapies for CNS disorders
- Where the technology is now, its evolution, achievements and pitfalls
- Competition
- Potential future applications
- Activity in the market, major players and winners
| Chapter 5 RNAi-based Therapies for Viral Infectious Diseases
- Key findings
- Introduction
- Respiratory Syncytial Virus
- Case study: ALN-RSV01 (Alnylam Pharmaceuticals)
- Hepatitis C
- Case study:TT-033 (Oncolys BioPharma/Pfizer/Tacere Therapeutics)
- Case study: miR-122 (Regulus Therapeutics)
- Case study: SPC3649 (Santaris Pharma A/S)
- Human Immunodeficiency Virus
- Case study: rHIV7-shl-TAR-CCR5RZ (Benitec Ltd/City of Hope)
- Pandemic Influenza
- Bioterrorism
- Other infectious diseases
- Conclusions
- Our opinion on RNAi-based Therapies for Viral Infectious Diseases
- Where the technology is now, its evolution, achievements and pitfalls
- Competition
- Potential future applications
- Activity in the market, major players and winners
Chapter 6 RNAi-based Therapies for Ocular Diseases
- Key findings
- Introduction
- Age-Related Macular Degeneration
- Case study: AGN-745 (Allergan/Sirna Therapeutics)
- Case study: VEGF-A165b sparing siRNA & Bevasiranib (Opko Health)
- Case study: PF-4523655 (Quark Pharmaceuticals/Pfizer)
- Diabetic Retinopathy & Diabetic Macular Edema
- Glaucoma
- Conclusions
- Our opinion on RNAi-based Therapies for Ocular Diseases
- Where the technology is now, its evolution, achievements and pitfalls
- Competition
- Potential future applications
- Activity in the market, major players and winners
Chapter 7 RNAi-based Therapies for Cardiovascular,
Metabolic & Other Conditions
- Key findings
- Introduction
- RNAi-based therapeutics for Cardiovascular Diseases
- Case study: miR-21 (Regulus Therapeutics)
- Case Study: MHC miRNA inhibitors (MiRagen Therapeutics Inc)
- RNAi-based Therapeutics for Metabolic Disorders
- Case Study: ApoB SNALP (Tekmira Pharmaceutical Corporation)
- Case study: MDR-04227 (MDRNA)
- Case Study: PCSK9 siRNA (Alnylam Pharmaceuticals)
- Case study: ApoB rxRNA and GeRP delivery (RXi Therapeutics)
- RNAi-based Therapeutics in Other Therapeutic Areas
- Case study: ALN-TTR siRNA (Alnylam Pharmaceuticals)
- Case study: HSP47 siRNA (Nitto Denko Technical Corporation)
- Case study: QPI-1002 (Silence Therapeutics/Quark Pharmaceuticals)
- Conclusion
- Our opinion on RNAi-based therapies for Cardiovascular, Metabolic & Other Diseases
- Where the technology is now, its evolution, achievements and pitfalls
- Competition
- Potential future applications
- Activity in the market, major players and winners
| Chapter 8 Challenges & Opportunities for RNAi-based Therapeutics & Diagnostics
- Key findings
- Introduction
- Stability & efficacy issues for RNAi research
- Stability
- Efficacy
- Safety issues for RNAi research
- Intellectual property
- Delivering RNAi-based therapies
- Challenges
- Opportunities
Chapter 9 The Future of RNAi-based Therapeutics & Diagnostics: Market Trends
- Key findings
- Key RNAi companies
- Leading RNAi-based therapeutic companies
- Leading miRNA-based therapeutic companies
- Leading RNAi-based diagnostic companies
- Recent deals & alliances
- The Global RNAi-based Therapy Market 2008-2014
- Analysis parameters
- Market forecast 2008-2014
- Global RNAi-based Therapy Market in 2020
- Drivers and market trends
Summary & Conclusions
Acknowledgements
Bibliography & Endnotes
This new report will enable you to...
- Assess the commercial potential of RNAi-based therapies in all of the key therapeutic areas: cancer, CNS disorders, viral infectious diseases, ocular diseases, plus cardiovascular, metabolic and other conditions
- Identify key pharma and delivery specialists advancing RNAi-based therapeutics and diagnostics
- Gain an in-depth understanding of the technological and delivery issues that companies developing RNAi-based therapies face
- Evaluate the options available for chemically and structurally modifying RNAi-silencing agents and delivering them to target tissues now and in the future
- Discover which companies are tackling efficacy and safety issues, and are in a prime position to exploit new opportunities
- Analyse how the market will evolve over the next decade, highlighting key trends, opportunities and challenges.
The report answers keys questions such as...
- Which companies and academic institutes are actively involved in RNAi-based therapeutic research?
- What are the key areas of therapeutic focus for RNAi-based therapeutics in the near and long-term?
- What are the key obstacles companies and delivery technologists need to overcome to commercialize RNAi-derived therapies?
- How will delivery technologies drive the development of innovative RNAi-based therapies in the future?
- Which RNAi technologies and agents are likely to win in the near-term and long-term, and why?
- Where are the market opportunities now and in the future?
- When are RNAi-based medicines likely to reach the market?
- What do we predict will be the value of the RNAi-based therapeutics by 2015 and 2020?
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