Home | Publications | Drug Discovery place holder
place holder place holder

Drug Discovery

The Outlook For RNAi

Accelerating drug discovery and the development of RNAi therapeutics

Publication Date   January 2005
Publisher   Business Insights
Product Type   Report
Pages   205
ISBN Number   not applicable
Product Code   RBI075
Ask a question about this product?

Price £1,495.00

approximately: $2,793 | €1,896

Summary


While the human genome project has provided vast amounts of sequence information, the in vivo functional analysis of thousands of genes has presented a significant challenge to researchers and to pharmaceutical companies in the discovery of new drug targets. However, RNAi-based screens have provided new opportunities for the discovery and validation of novel therapeutic targets in several disease areas such as cancer and infectious diseases.

Content


  • Executive Summary
    • Current Rna Technologies
    • Design, Production And Delivery Of Rnai
    • The Future Of Rnai In Research And Drug Discovery
    • The Future Of Rnai Drug Therapies
    • Emerging Rna Technologies And Future Trends
    • Patents And Strategic Alliances In Rnai Technology
    • Rnai Markets And Trends
  • Chapter 1 Current Rna Technologies
    • Summary
    • Introduction
    • History Of Rnai
    • From Dna To Rna To Proteins
    • Mrna Regulation
    • Gene Expression
    • Antisense Technology
    • Oligonucleotides (Ogns)
    • Peptide Nuclei Acids (Pnas)
    • Locked Nucleic Acids (Lna)
    • Triple Helix Dna Or Triple Helix-Forming Oligonucleotides (Tfos)
    • Ribozymes
    • Dnazymes
    • Aptamers
    • Rna Interference
    • Sirnas Versus Dsrna
    • Sirnas Versus Shrna
    • Conclusions
  • Chapter 2 Design, Production And Delivery Of Rnai
    • Summary
    • Introduction
    • Cost-Effective Rna Design
    • Cost-Effective Synthesis Of Sirna
    • Chemical Synthesis
    • Conclusions
    • In Vitro Transcription
    • Dicer Reaction
    • Expression Vectors
    • Dna-Directed Rnai (Ddrnai)
    • Expressed Interfering Rna (Eirna)
    • Conclusions
    • Improvements In Sirna Stability
    • Chemical Modifications
    • Formulation Modifications
    • Small Molecule Conjugation
    • Synthetic Vector Systems
    • Conclusion
    • Rnai Delivery Options
    • Viral Vectors
    • Conclusions
  • Chapter 3 The Future Of Rnai In Research And Drug Discovery
    • Summary
    • Introduction
    • Applications Of Rnai In Research
    • Functional Genomics
    • Signaling Pathways
    • Applications Of Rnai In Drug Discovery
    • Gene Expressions Studies
    • Target Validation
    • Toxicogenomics
    • Applications Of Rnai In Drug Development
    • Transgenics
    • The Impact Of Rnai In R&d
  • Chapter 4 The Future Of Rnai Drug Therapies
    • Summary
    • Introduction
    • Shift From Antisense To Rnai
    • Ocular Diseases
    • Age-Related Macular Degeneration (Amd)
    • Key Rnai Players
    • Diabetic Retinopathy (Dr)
    • Key Rnai Players
    • Conclusions
    • Infectious Diseases
    • Hepatitis C Virus (Hcv)
    • Key Rnai Players
    • Hiv
    • Cmv (Cytomegalovirus)
    • Key Rnai Players
    • Conclusions
    • Respiratory
    • Respiratory Syncytial Virus (Rsv)
    • Key Rnai Players
    • Asthma
    • Key Rnai Players
    • Cystic Fibrosis
    • Key Rnai Players
    • Conclusions
    • Neurological Diseases
    • Huntingdon's Disease (Hd)
    • Key Rnai Players
    • Amyotrophic Lateral Sclerosis (Als Or Lou Gehrig's Disease)
    • Key Rnai Players
    • Spinal Cord Injury (Sci)
    • Key Rnai Players
    • Parkinson's Disease (Pd)
    • Alzheimer's Disease (Ad)
    • Pain
    • Conclusions
    • Oncology
    • Angiogenesis
    • Key Rnai Players
    • Oncogenes
    • Key Rnai Players
    • Drug Resistance And Enhancement
    • Key Rnai Players
    • Conclusions On Rnai In Oncology
    • Cardiovascular Diseases
    • Key Rnai Players
    • Conclusions
    • Metabolic Disorders
    • Diabetes
    • Key Rnai Players
    • The Future Role Of Rnai-Based Therapeutics
  • Chapter 5 Emerging Rna Technologies And Future Trends
    • Summary
    • Introduction
    • Second Generation Sirnas
    • Multifunctional Sirnas
    • Hyperfunctional Or Superactive Sirnas
    • No-Ribose Small Inhibitory Nucleic Acids (Sinas)
    • Sirnas Conjugated With Small Molecule Drugs
    • Alternative Rna Based Therapies:
    • Micro Rnas (Mirnas)
    • Mirna Processing
    • Mirna In Embryonic Development
    • Mirna In Neurological Disorders
    • Mirna In Cancer
    • Future Direction Of Mirna Research
    • Small Nucleolar Rnas (Snornas)
    • Aptamers
  • Chapter 6 Patents And Strategic Alliances In Rnai Technology
    • Summary
    • Introduction
    • Patents For Sirna Reagents
    • Patents For Sirna Therapeutics
    • Alnylam Pharmaceuticals (Cambridge, Ma, Us)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Benitec Ltd (Queensland, Australia)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Sirna Therapeutics (Formerly Ribozyme Pharmaceuticals)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Acuity Pharmaceuticals (Philadelphia, Pa, Us)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Atugen Ag (Dresden, Germany)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Cytrx Labs (Los Angeles, Ma, USA)
    • Patent Position
    • Strategic Alliances, 2003-2005
    • Intradigm (Rockville, Md, USA)
    • Nucleonics Inc. (Horsham, Pa, USA)
    • Future Impact Of Ip On Rnai Research
  • Chapter 7 Rnai Markets And Trends
    • Summary
    • Introduction
    • The Rnai Market
    • Market Size And Future Trends
    • Sirna Synthesis And Delivery
    • Rnai Reagents
    • Rnai In Drug Discovery And Target Validation
    • Rnai Therapeutics
  • Chapter 8 Appendix
    • Acknowledgements
    • Index
    • Bibliography
    • Glossary
    • References
  • List Of Figures
    • Figure 1.1: History Of Rnai
    • Figure 1.2: Schematic Of Dna, Genes And Proteins
    • Figure 1.3: Schematic Of Gene Splicing
    • Figure 1.4: Major Mechanisms For Antisense Ogn Action
    • Figure 1.5: Mechanism Of Preventing Translation Using Ogn Technology
    • Figure 1.6: Chemical Structure Of Pna Versus Dna
    • Figure 1.7: Chemical Structure Of Lna Versus Rna
    • Figure 1.8: Mechanism Of Preventing Translation Using Triple Helix Dna Technology
    • Figure 1.9: Mechanism Of Preventing Translation Using Ribozymes
    • Figure 1.10: Schematic Of The Mechanism Of Gene Silencing By Rnai
    • Figure 1.11: Schematic Of The Mechanism Of Shrnas
    • Figure 2.12: Advantages And Disadvantages Of Sirna Synthesis Methods
    • Figure 2.13: In Vitro Transcription Of Sirnas
    • Figure 2.14: Dicer Digestion Of Dsrnas
    • Figure 2.15: Psirna Plasmid Vector System
    • Figure 2.16: Mechanism Of Ddrnai
    • Figure 2.17: Chemical Modifications Of Sirnas Increase Stability And Pk
    • Figure 2.18: Chol- Sirnas Improve Tissue Uptake And Pk
    • Figure 2.19: Intradigm's Nano-Delivery Technology Targetran
    • Figure 2.20: Summary Of Viral Vector Advantages And Disadvantages
    • Figure 3.21: The Application Of Tca In Gene Expression
    • Figure 3.22: Optimization Of Lead Compounds With Sirnas
    • Figure 3.23: Comparison Of Gene Expression Profiles To Optimize Lead Compounds
    • Figure 3.24: Investigation Of The Intracellular Mechanism Of Endothelin A Receptor
    • Figure 3.25: Schematic Of Knock-Out And Knock-Down Transgenics
    • Figure 3.26: Heritable Suppression Of Neil-1 In Mouse Model
    • Figure 3.27: Artemicetm Rnai In Vivo In 4 Months
    • Figure 3.28: Artemis Pharmaceutical Timelines For Transgenic Animals
    • Figure 3.29: Status Leptinr Knockdown Using Shrnas
    • Figure 3.30: Impact Of Rnai In R&d
    • Figure 4.31: Antisense Drugs Currently In Clinical Development
    • Figure 4.32: Rnai Drugs Currently In Clinical Development
    • Figure 4.33: Development Of Amd
    • Figure 4.34: Sirna Targeting Vegf Reduces Blood Vessel Growth In The Cornea
    • Figure 4.35: Lead Sirna Candidates Block Hcv Replication
    • Figure 4.36: Hcv Target Destruction In Mouse Liver
    • Figure 4.37: Efficacy Of Hiv Drug In Vitro
    • Figure 4.38: In Vivo Efficacy Of Direct Rnai For Rsv
    • Figure 4.39: Systemic Sirna Leads To Significant Reduction In Apolipoproteins
    • Figure 5.40: Conventional Risc Silencing Pathways And Risc Pathway Using "On-Target" Sirna Reagents
    • Figure 5.41: Sirna Risc Process Using "On-Target Plus" Sirna Reagents
    • Figure 5.42: Schematic Representation Of Aptazyme Development
    • Figure 6.43: Key Rna-Based Companies Targeting Rnai Reagents
    • Figure 6.44: Key Rna-Based Companies Targeting Therapeutic Agents
    • Figure 6.45: Sirna Therapeutics' Ip Portfolio And Therapeutic Areas
    • Figure 7.46: Rnai Market Segments, 2004
    • Figure 7.47: Growth In The Rnai Market 2004-2010
    • Figure 7.48: Alliances In Rnai R&d
    • Figure 7.49: Potential Value Of Therapy Areas Targeted By Rnai Therapeutics, 2004 & 2010
  • List Of Tables
    • Table 1.1: Advantages And Disadvantages Of Ogn Technology
    • Table 1.2: Advantages And Disadvantages Of Modified Ogns
    • Table 1.3: Advantages And Disadvantages Of Tfos
    • Table 1.4: Advantages And Disadvantages Of Ribozymes
    • Table 1.5: Advantages And Disadvantages Of Dnazymes
    • Table 1.6: Advantages And Disadvantages Of Aptamers
    • Table 1.7: Genes Crucial For Rnai In Model Organisms
    • Table 1.8: Advantages Of Rnai
    • Table 1.9: Disadvantages Of Rnai
    • Table 2.10: Algorithms Available For Designing Sirnas
    • Table 2.11: Class Of Functional Rna Molecule
    • Table 2.12: Companies Offering Sirna Synthesis
    • Table 2.13: Advantages Of Ddrnai Versus Sirna
    • Table 2.14: Advantages Of Eirna Versus Sirnas
    • Table 3.15: Commercial Sirna Libraries
    • Table 4.16: Antiviral Sirna Targets
    • Table 4.17: Rnai-Based Targeted Therapies
    • Table 4.18: Examples Of Rnai Targets For Neuronal Pain
    • Table 4.19: Chemotherapeutic Sirna Targets
    • Table 5.20: Animal Mirna Genes With Genetically Assigned Functions
    • Table 6.21: Rna Patents Registered Worldwide Up To March 2005
    • Table 7.22: Companies Involved In Rnai Technologies, A-M
    • Table 7.23: Companies Involved In Rnai Technologies, N-Z
    • Table 7.24: Sales Forecasts For Total Rnai Market, 2004-2015
    • Table 7.25: Sales Forecasts For Sirna Synthesis And Delivery, 2004-2015
    • Table 7.26: Sales Forecasts For Rnai Reagents, 2004-2015
    • Table 7.27: Sales Forecasts For Rnai In Drug Discovery & Target Validation, 2004-2015
    • Table 7.28: Sales Forecasts For Rnai Therapeutics, 2004-2015
    • Table 7.29: Sales Forecasts For Rnai Therapeutic Drugs Launched 2010-2015 188