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The Future of RNAi Therapeutics: Drug Pipelines and Prospects
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Features of this market research: | 253 pages | |||||||||
| About this market survey: |
""The total RNAi therapy market is estimated to value $580m by 2012. This forecast is based upon current treatments, levels of market satisfaction and five new Phase II/III RNAi agents profiled in thi.....
""The total RNAi therapy market is estimated to value $580m by 2012. This forecast is based upon current treatments, levels of market satisfaction and five new Phase II/III RNAi agents profiled in this report."" RNA interference continues to offer significant promise for a host of therapeutic treatments. Potentially any disease-causing gene, cell type or tissue can be targeted with RNAi, including those not ‘druggable’ with small molecules or protein-based therapies. Pharma companies are increasingly investing in siRNA optimization or examining siRNA alternatives, in addition to staking out proprietary positions in lipid-based, nanotransporter-based and alternative delivery technologies. The development and licensing of such platforms will become crucial for developers over the coming years. ‘The Future of RNAi Therapeutics: Drug pipelines and prospects’ is a report published by Business Insights that provides an in-depth assessment of recent progress and future opportunities for RNAi players in major therapeutic areas including infectious diseases, cancer, inflammation/immune dysfunction, CNS disorders and cardiology/metabolism. The latest trends in RNAi therapeutics are examined, focusing on the main areas of clinical need, general/proprietary technologies and company pipelines, and the activities and strategies of 37 RNAi-based developers are profiled. This report also provides forecasts for market growth to 2012 across major RNAi market segments. Identify the developmental status of key RNAi agents, review the latest advances in RNAi technologies and assess the competitive landscape with this report’s analysis of 37 companies across major therapeutic areas. ‘The Future of RNAi Therapeutics’ is a report published by Business Insights that provides an in-depth assessment of recent progress and future opportunities for RNAi players in major therapeutic areas including infectious diseases, cancer, inflammation/immune dysfunction, CNS disorders and cardiology/metabolism. The latest trends in RNAi therapeutics are examined, focusing on the main areas of clinical need, proprietary technologies and company pipelines, and the activities and strategies of 37 RNAi-based developers are profiled. This report also provides forecasts for market growth to 2012 across major RNAi market segments. Measure the developmental progress of 97 RNAi agents, review the latest advances in delivery technologies and evaluate 37 RNAi-based companies across major therapeutic areas... Use this report to... Analyze emerging RNAi agents and delivery technologies and examine new molecular targets and pipeline antisense drugs. Evaluate 97 RNAi compounds in development for infectious and inflammatory diseases, cancer and other major disease categories. Assess 37 competitors and potential collaborators developing RNAi technologies and examine the latest RNAi-related patenting trends. Identify potential growth segments and develop future strategies with this report’s forecasts for the global RNAi therapy market to 2012. Report Highlights [Fade out the market survey infos] |
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RNAi Therapeutics: Drug pipelines and prospects Executive Summary 14 RNAi in Infectious Disease 15 RNAi in Cancer 16 RNAi in Inflammatory and Other Diseases 18 RNAi Companies: Technologies and Therapies 19 Market Trends and Opportunities 19 Chapter 1 Introduction 24 Summary 24 Introduction 25 Antisense technology 35 Antisense oligonucleotides 35 Ribozymes and DNAzymes 37 Discovery of natural RNAi activated by dsRNA 37 Mechanisms of RNAi stimulated by dsRNA 38 Approaches to activate RNAi in mammals 39 Discovery of micro-RNAs 40 The potential for RNAi-based therapeutics 41 The promise of siRNA therapeutics 41 The promise of miRNAs therapeutics 43 Optimizing siRNA design 44 On- and off-target activity 44 Immune system reactivity 46 Improving siRNA stability 47 Optimizing siRNA delivery 48 Naked siRNA 49 Liposomes and lipoplexes 50 Polymer carriers/nanoparticles 51 Conjugates 52 Optimizing shRNA delivery 53 Chapter 2 RNAi in infectious diseases 56 Summary 56 Introduction 57 Respiratory viral infections 58 RSV virus 60 Clinical and biological features 60 Design of RNAi therapeutics 62 RNAi therapeutics in development 63 Influenza virus 64 Clinical and biological features 65 Design of RNAi therapeutics 68 RNAi therapeutics in development 69 SARS 70 RNAi therapeutics in development 71 Chronic viral infections 71 Hepatitis viruses 71 Clinical and biological features 71 Design of RNAi therapeutics 73 RNAi drugs in development 74 HIV virus 77 Clinical and biological features 77 Design of RNAi therapeutics 80 RNAi drugs in development 82 HPV 83 HSV 83 Viral hemorrhagic fever and Ebola 84 Malaria 85 Chapter 3 RNAi in Cancer 88 Summary 88 Introduction 89 Cancer types 91 Treatment of cancer 94 Approaches to RNAi in cancer therapy 96 Approaches to RNAi delivery 99 Targeting microRNAs 100 RNAi cancer therapeutics in development 100 Agents directed at established targets 100 Agents directed at novel targets 102 Agents directed at multiple targets 104 Agents directed at unspecified targets 105 microRNAs 105 DNAi therapeutics 105 Chapter 4 RNAi in inflammatory and other diseases 108 Summary 108 Introduction 109 Inflammation/immune dysfunction 109 Current therapies 112 Allergies and respiratory diseases 114 RNAi therapeutics in development 116 Age-related macular degeneration 118 RNAi therapeutics in development 120 Other inflammatory conditions 122 RNAi therapeutics in development 123 Acute Renal Failure 123 RNAi therapeutics in development 124 CNS diseases 125 Huntington's disease 125 RNAi therapeutics in development 125 Parkinson's disease 126 RNAi therapeutics in development 126 Other CNS conditions 127 Cardiology and metabolism 129 Cardiovascular disease 129 RNAi agents in development 130 Type 2 diabetes 132 RNAi agents in development 132 Non-alcoholic steatohepatitis 133 Other indications 133 Chapter 5 RNAi Companies: Technologies and Therapies 138 Summary 138 Alnylam Pharmaceuticals Inc 139 Company description 139 RNAi-related technology 139 Candidate RNAi therapeutics 141 RNAi-related agreements 142 Ambrilia Biopharma Inc 145 Company description 145 RNAi-related technology 146 Candidate RNAi therapeutics 146 Angioblast Systems Inc 146 Company description 146 RNAi-related technology 146 Candidate RNAi therapeutics 146 AVI BioPharma Inc 147 Company description 147 Exon Skipping Pre-RNA Interference Technology (ESPRIT) 148 RNA splicing-related agreements 148 Benitec Ltd 149 Company description 149 RNAi-related technology 149 Candidate RNAi therapeutics 150 RNAi-related agreements 150 BioCancell Therapeutics Inc 151 Company description 151 Candidate RNAi therapeutics 152 RNAi-related agreements 152 Calando Pharmaceuticals Inc 152 Company description 152 RNAi-related technology 153 Candidate RNAi therapeutics 153 RNAi-related agreements 153 Cequent Pharmaceuticals Inc 154 Company description 154 RNAi-related technology 154 Candidate RNAi therapeutics 154 Dicerna Pharmaceuticals Inc 154 Company description 154 RNAi-related technology 155 Candidate RNAi therapeutics 155 Generex Biotechnology Corp 155 Company description 156 RNAi-related technology 156 Candidate RNAi therapeutics 156 RNAi-related agreements 156 Genesis Research and Development Corp 157 Company description 157 RNAi-related technology 157 Candidate RNAi therapeutics 158 Green Cross Corp 158 Company description 158 Candidate RNAi therapeutics 158 Gruenenthal Group 159 Company description 159 RNAi-related technology 159 Candidate RNAi therapeutics 159 Intradigm Corp 159 Company description 159 RNAi-related technology 159 Candidate RNAi therapeutics 160 RNAi-related agreements 160 Isis Pharmaceuticals Inc 161 Company description 161 RNAi-related agreements 162 Lorus Therapeutics Inc 163 Company description 163 Candidate RNAi therapeutics 163 MDRNA Inc 164 Company description 164 RNAi-related technology 164 Candidate RNAi therapeutics 165 RNAi-related agreements 165 Merck & Co Inc 165 Company description 165 RNAi-related technology 166 Candidate RNAi therapeutics 166 RNAi-related agreements 167 Nucleonics Inc 168 Company description 168 RNAi-related technology 168 Candidate RNAi therapeutics 169 RNAi-related agreements 169 Opko Health Inc 169 Company description 169 Candidate RNAi therapeutics 170 RNAi-related agreements 170 ProNAi Therapeutics Inc 170 Company description 170 RNAi-related technology 171 Candidate DNAi therapeutics 171 DNAi-related agreements 171 Quark Pharmaceuticals Inc 172 Company description 172 RNAi-related technology 172 Candidate RNAi therapeutics 172 RNAi-related agreements 172 Regulus Therapeutics LLC 173 Company description 173 miRNA-related technology 173 Candidate miRNA therapeutics 174 RNAi-related agreements 174 Rosetta Genomics Ltd 175 Company description 175 miRNA-related technology 175 Candidate miRNA therapeutics 175 miRNA-related agreements 176 RXi Pharmaceuticals Corp 176 Company description 176 RNAi-related technology 177 Candidate RNAi therapeutics 177 RNAi-related agreements 177 Santaris Pharma A/S 178 Company description 178 RNAi-related technology 178 Candidate miRNA therapeutics 179 RNAi-related agreements 179 Senesco Technologies Inc 180 Company description 180 Candidate RNAi therapeutics 180 Senetek plc 181 Company description 181 Candidate RNAi therapeutics 181 RNAi-related agreements 181 Silence Therapeutics plc 182 Company description 182 RNAi-related technology 182 Candidate RNAi therapeutics 183 RNAi-related agreements 183 Sirnaomics Inc 184 Company description 184 RNAi-related technology 184 Candidate RNAi therapeutics 184 RNAi-related agreements 184 siRNAsense AS 185 Company description 185 Candidate RNAi therapeutics 185 RNAi-related agreements 185 Stelic Institute & Co 186 Company description 186 Candidate RNAi therapeutics 186 RNAi-related agreements 186 Targeted Genetics Corp 187 Company description 187 RNAi-related technology 187 Candidate RNAi therapeutics 188 RNAi-related agreements 188 Tekmira Pharmaceuticals Corp 188 Company description 188 RNAi-related technology 189 Candidate RNAi therapeutics 189 RNAi-related agreements 189 ToleroTech Inc 190 Company description 190 RNAi-related technology 190 Candidate RNAi therapeutics 191 Topigen Pharmaceuticals Inc 191 Company description 191 RNAi-related technology 191 Candidate RNAi therapeutics 192 TransDerm Inc 192 Company description 192 RNAi-related technology 192 Candidate RNAi therapeutics 193 Chapter 6 Market trends and opportunities 196 Summary 196 Validating RNAi in the clinic 197 From first-in-class to best-in-class 199 Exploring alternatives to siRNAs 200 Overcoming delivery challenges 201 Moving beyond ""druggable"" targets 202 Intellectual property considerations 207 Introduction to RNAi patents 207 Fire and Mello Patent 208 Tuschl Patents 208 Kreutzer-Limmer Patents 210 Benitec patents 210 Crooke / RNAi modification patents 212 Glover patent 213 RNAi target patents 213 miRNA patents 214 Effect of patents on infringing R&D 214 Patent survey results 214 Market value considerations 225 Commercial prospects 225 Summary of key indications 226 Forecasts 226 Wet AMD: bevasiranib and others 226 RSV infection: ALN-RSV01 228 Acute renal failure: AKTi-5 228 The RNAi bottom line 229 Company details 232 Alnylam Pharmaceuticals Inc 232 Ambrilia Biopharma Inc 232 Angioblast Systems Inc 233 AVI BioPharma Inc 233 Benitec Ltd 234 BioCancell Therapeutics Inc 234 Calando Pharmaceuticals Inc 235 Cequent Pharmaceuticals Inc 235 Dicerna Pharmaceuticals Inc 236 Generex Biotechnology Corp 236 Genesis Research and Development Corp 237 Green Cross Corp 237 Gruenenthal Group 238 Intradigm Corp 238 Isis Pharmaceuticals Inc 238 Lorus Therapeutics Inc 239 MDRNA Inc 239 Merck & Co Inc 240 Nucleonics Inc 240 Opko Health Inc 241 ProNAi Therapeutics Inc 241 Quark Pharmaceuticals Inc 242 Regulus Therapeutics LLC 242 Rosetta Genomics Ltd 242 RXi Pharmaceuticals Corp 243 Santaris Pharma A/S 243 Senesco Technologies Inc 244 Senetek plc 244 Silence Therapeutics plc 245 Sirnaomics Inc 245 siRNAsense AS 246 Stelic Institute & Co 246 Targeted Genetics Corp 246 Tekmira Pharmaceuticals Corp 247 ToleroTech Inc 247 Topigen Pharmaceuticals Inc 248 TransDerm Inc 248 Glossary 249 Abbreviations and acronyms 249 [Fade out table of contents] |
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Table 1.1: RNAi Oligonucleotide Indications – Infectious Disease 27 Table 1.2: RNAi Oligonucleotide Indications – Cancer 28 Table 1.3: RNAi Oligonucleotide Indications – Cancer (continued) 29 Table 1.4: RNAi Oligonucleotide Indications – Cancer (continued) 30 Table 1.5: RNAi Oligonucleotide Indications – Miscellaneous Disorders 30 Table 1.6: RNAi Oligonucleotide Indications – CNS 31 Table 1.7: RNAi Oligonucleotide Indications – Inflammation/Immune Disorders 32 Table 1.8: RNAi Oligonucleotide Indications – Inflammation/Immune Disorders (continued) 33 Table 1.9: RNAi Oligonucleotide Indications – Cardiology, Metabolism 34 Table 5.10: Financial Details, year ending Dec 2007, (US$m): Alnylam Pharmaceuticals Inc 139 Table 5.11: Financial Details, year ending 2007, (Can$m): Ambrilia Biopharma Inc 145 Table 5.12: Financial Details, year ending Dec 2007, (US$m): AVI BioPharma Inc 147 Table 5.13: Financial Details, year ending Jun 2007, (Aus$m): Benitec Ltd 149 Table 5.14: Financial Details, year ending Dec 2006, (NISm): BioCancell Therapeutics Inc 151 Table 5.15: Financial Details, year ending July 2007, (US$m): Generex Biotechnology Corp 155 Table 5.16: Financial Details, year ending Dec 2007, (NZ$m): Genesis Research and Development Corp 157 Table 5.17: Financial Details, year ending Dec 2006, (?m): Green Cross Corp 158 Table 5.18: Financial Details, year ending Dec 2007, (US$m): Isis Pharmaceuticals Inc 161 Table 5.19: Financial Details, year ending May 2007, (Can$m): Lorus Therapeutics Inc 163 Table 5.20: Financial Details, year ending Dec 2007, (US$m): MDRNA Inc 164 Table 5.21: Financial Details, year ending Dec 2007, (US$m): Merck & Co Inc 165 Table 5.22: Financial Details, year ending Dec 2007, (US$m): Opko Health Inc 169 Table 5.23: Financial details, year ending Dec 2007, (US$m): Rosetta Genomics Ltd 175 Table 5.24: Financial details, year ending Dec 2007, (US$m):RXi Pharmaceuticals Corp 176 Table 5.25: Financial details, year ending Jun 2007, (US$m): Senesco Technologies Inc 180 Table 5.26: Financial details, year ending Dec 2007, (US$m): Senetek plc 181 Table 5.27: Financial details, year ending Dec 2007, (£m): Silence Therapeutics plc 182 Table 5.28: Financial details, year ending Dec 2007, (US$m): Targeted Genetics Corp 187 Table 5.29: Financial details, year ending Dec 2007, (Can$m): Tekmira Pharmaceuticals Corp 188 Table 6.30: Common RNAi/Antisense Targets 204 Table 6.31: Common RNAi/Antisense Targets (Contd.) 205 Table 6.32: Targets of Antisense Oligonucleotides but not RNAis 206 Table 6.33: WIPO RNAi Patents: Filing and Publication Dates 215 Table 6.34: USPTO RNAi Patents: Filing and Publication Dates 215 Table 6.35: WIPO RNAi Patents: Top Assignees 217 Table 6.36: WIPO RNAi Patents: Top Assignees (Contd.) 218 Table 6.37: WIPO RNAi Patents: Top Assignees (Contd.) 219 Table 6.38: USPTO RNAi Patents: Top Assignees 220 Table 6.39: Leading RNAi Oligonucleotide Originators 221 Table 6.40: Key data: Forecasts for wet AMD, RSV therapy and acute renal failure ($m), 2012 229 Figure 6.1: RNAi by development stage 197 Figure 6.2: RNAi by origin of material 203 Figure 6.3: RNAi USPTO patents: therapy area 222 Figure 6.4: RNAi WIPO patents: therapy area 223 Figure 6.5: RNAi USPTO Patents: patent focus 224 Figure 6.6: RNAi WIPO patents: patent focus 225 [Fade out list of tables / figures] |
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