Biotechnology
Future of RNAi Therapeutics, The
Drug Pipelines and Prospects
| Publication Date | September 2008 |
| Publisher | Business Insights |
| Product Type | Report |
| Pages | 253 |
| ISBN Number | not applicable |
| Product Code | RBI00185 |
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Summary
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 new 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.
Contents
- RNAi Therapeutics: Drug pipelines and prospects
- Executive Summary
- RNAi in Infectious Disease
- RNAi in Cancer
- RNAi in Inflammatory and Other Diseases
- RNAi Companies: Technologies and Therapies
- Market Trends and Opportunities
- Chapter 1 Introduction
- Summary
- Introduction
- Antisense technology
- Antisense oligonucleotides
- Ribozymes and DNAzymes
- Discovery of natural RNAi activated by dsRNA
- Mechanisms of RNAi stimulated by dsRNA
- Approaches to activate RNAi in mammals
- Discovery of micro-RNAs
- The potential for RNAi-based therapeutics
- The promise of siRNA therapeutics
- The promise of miRNAs therapeutics
- Optimizing siRNA design
- On- and off-target activity
- Immune system reactivity
- Improving siRNA stability
- Optimizing siRNA delivery
- Naked siRNA
- Liposomes and lipoplexes
- Polymer carriers/nanoparticles
- Conjugates
- Optimizing shRNA delivery
- Chapter 2 RNAi in infectious diseases
- Summary
- Introduction
- Respiratory viral infections
- RSV virus
- Clinical and biological features
- Design of RNAi therapeutics
- RNAi therapeutics in development
- Influenza virus
- Clinical and biological features
- Design of RNAi therapeutics
- RNAi therapeutics in development
- SARS
- RNAi therapeutics in development
- Chronic viral infections
- Hepatitis viruses
- Clinical and biological features
- Design of RNAi therapeutics
- RNAi drugs in development
- HIV virus
- Clinical and biological features
- Design of RNAi therapeutics
- RNAi drugs in development
- HPV
- HSV
- Viral hemorrhagic fever and Ebola
- Malaria
- Chapter 3 RNAi in Cancer
- Summary
- Introduction
- Cancer types
- Treatment of cancer
- Approaches to RNAi in cancer therapy
- Approaches to RNAi delivery
- Targeting microRNAs
- RNAi cancer therapeutics in development
- Agents directed at established targets
- Agents directed at novel targets
- Agents directed at multiple targets
- Agents directed at unspecified targets
- microRNAs
- DNAi therapeutics
- Chapter 4 RNAi in inflammatory and other diseases
- Summary
- Introduction
- Inflammation/immune dysfunction
- Current therapies
- Allergies and respiratory diseases
- RNAi therapeutics in development
- Age-related macular degeneration
- RNAi therapeutics in development
- Other inflammatory conditions
- RNAi therapeutics in development
- Acute Renal Failure
- RNAi therapeutics in development
- CNS diseases
- Huntington's disease
- RNAi therapeutics in development
- Parkinson's disease
- RNAi therapeutics in development
- Other CNS conditions
- Cardiology and metabolism
- Cardiovascular disease
- RNAi agents in development
- Type 2 diabetes
- RNAi agents in development
- Non-alcoholic steatohepatitis
- Other indications
- Chapter 5 RNAi Companies: Technologies and Therapies
- Summary
- Alnylam Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Ambrilia Biopharma Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Angioblast Systems Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- AVI BioPharma Inc
- Company description
- Exon Skipping Pre-RNA Interference Technology (ESPRIT)
- RNA splicing-related agreements
- Benitec Ltd
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- BioCancell Therapeutics Inc
- Company description
- Candidate RNAi therapeutics
- RNAi-related agreements
- Calando Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Cequent Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Dicerna Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Generex Biotechnology Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Genesis Research and Development Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Green Cross Corp
- Company description
- Candidate RNAi therapeutics
- Gruenenthal Group
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Intradigm Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Isis Pharmaceuticals Inc
- Company description
- RNAi-related agreements
- Lorus Therapeutics Inc
- Company description
- Candidate RNAi therapeutics
- MDRNA Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Merck & Co Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Nucleonics Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Opko Health Inc
- Company description
- Candidate RNAi therapeutics
- RNAi-related agreements
- ProNAi Therapeutics Inc
- Company description
- RNAi-related technology
- Candidate DNAi therapeutics
- DNAi-related agreements
- Quark Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Regulus Therapeutics LLC
- Company description
- miRNA-related technology
- Candidate miRNA therapeutics
- RNAi-related agreements
- Rosetta Genomics Ltd
- Company description
- miRNA-related technology
- Candidate miRNA therapeutics
- miRNA-related agreements
- RXi Pharmaceuticals Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Santaris Pharma A/S
- Company description
- RNAi-related technology
- Candidate miRNA therapeutics
- RNAi-related agreements
- Senesco Technologies Inc
- Company description
- Candidate RNAi therapeutics
- Senetek plc
- Company description
- Candidate RNAi therapeutics
- RNAi-related agreements
- Silence Therapeutics plc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Sirnaomics Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- siRNAsense AS
- Company description
- Candidate RNAi therapeutics
- RNAi-related agreements
- Stelic Institute & Co
- Company description
- Candidate RNAi therapeutics
- RNAi-related agreements
- Targeted Genetics Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- Tekmira Pharmaceuticals Corp
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- RNAi-related agreements
- ToleroTech Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Topigen Pharmaceuticals Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- TransDerm Inc
- Company description
- RNAi-related technology
- Candidate RNAi therapeutics
- Chapter 6 Market trends and opportunities
- Summary
- Validating RNAi in the clinic
- From first-in-class to best-in-class
- Exploring alternatives to siRNAs
- Overcoming delivery challenges
- Moving beyond "druggable" targets
- Intellectual property considerations
- Introduction to RNAi patents
- Fire and Mello Patent
- Tuschl Patents
- Kreutzer-Limmer Patents
- Benitec patents
- Crooke / RNAi modification patents
- Glover patent
- RNAi target patents
- miRNA patents
- Effect of patents on infringing R&D
- Patent survey results
- Market value considerations
- Commercial prospects
- Summary of key indications
- Forecasts
- Wet AMD: bevasiranib and others
- RSV infection: ALN-RSV01
- Acute renal failure: AKTi-5
- The RNAi bottom line
- Company details
- Alnylam Pharmaceuticals Inc
- Ambrilia Biopharma Inc
- Angioblast Systems Inc
- AVI BioPharma Inc
- Benitec Ltd
- BioCancell Therapeutics Inc
- Calando Pharmaceuticals Inc
- Cequent Pharmaceuticals Inc
- Dicerna Pharmaceuticals Inc
- Generex Biotechnology Corp
- Genesis Research and Development Corp
- Green Cross Corp
- Gruenenthal Group
- Intradigm Corp
- Isis Pharmaceuticals Inc
- Lorus Therapeutics Inc
- MDRNA Inc
- Merck & Co Inc
- Nucleonics Inc
- Opko Health Inc
- ProNAi Therapeutics Inc
- Quark Pharmaceuticals Inc
- Regulus Therapeutics LLC
- Rosetta Genomics Ltd
- RXi Pharmaceuticals Corp
- Santaris Pharma A/S
- Senesco Technologies Inc
- Senetek plc
- Silence Therapeutics plc
- Sirnaomics Inc
- siRNAsense AS
- Stelic Institute & Co
- Targeted Genetics Corp
- Tekmira Pharmaceuticals Corp
- ToleroTech Inc
- Topigen Pharmaceuticals Inc
- TransDerm Inc
- Glossary
- Abbreviations and acronyms
- List of Figures
- Figure 6.1: RNAi by development stage
- Figure 6.2: RNAi by origin of material
- Figure 6.3: RNAi USPTO patents: therapy area
- Figure 6.4: RNAi WIPO patents: therapy area
- Figure 6.5: RNAi USPTO Patents: patent focus
- Figure 6.6: RNAi WIPO patents: patent focus
- Table 1.1: RNAi Oligonucleotide Indications - Infectious Disease
- Table 1.2: RNAi Oligonucleotide Indications - Cancer
- Table 1.3: RNAi Oligonucleotide Indications - Cancer (continued)
- Table 1.4: RNAi Oligonucleotide Indications - Cancer (continued)
- Table 1.5: RNAi Oligonucleotide Indications - Miscellaneous Disorders
- Table 1.6: RNAi Oligonucleotide Indications - CNS
- Table 1.7: RNAi Oligonucleotide Indications - Inflammation/Immune Disorders
- Table 1.8: RNAi Oligonucleotide Indications - Inflammation/Immune Disorders (continued)
- Table 1.9: RNAi Oligonucleotide Indications - Cardiology, Metabolism
- Table 5.10: Financial Details, year ending Dec 2007, (US$m): Alnylam Pharmaceuticals Inc
- Table 5.11: Financial Details, year ending 2007, (Can$m): Ambrilia Biopharma Inc
- Table 5.12: Financial Details, year ending Dec 2007, (US$m): AVI BioPharma Inc
- Table 5.13: Financial Details, year ending Jun 2007, (Aus$m): Benitec Ltd
- Table 5.14: Financial Details, year ending Dec 2006, (NISm): BioCancell Therapeutics Inc
- Table 5.15: Financial Details, year ending July 2007, (US$m): Generex Biotechnology Corp
- Table 5.16: Financial Details, year ending Dec 2007, (NZ$m): Genesis Research and Development Corp
- Table 5.17: Financial Details, year ending Dec 2006, (?m): Green Cross Corp
- Table 5.18: Financial Details, year ending Dec 2007, (US$m): Isis Pharmaceuticals Inc
- Table 5.19: Financial Details, year ending May 2007, (Can$m): Lorus Therapeutics Inc
- Table 5.20: Financial Details, year ending Dec 2007, (US$m): MDRNA Inc
- Table 5.21: Financial Details, year ending Dec 2007, (US$m): Merck & Co Inc
- Table 5.22: Financial Details, year ending Dec 2007, (US$m): Opko Health Inc
- Table 5.23: Financial details, year ending Dec 2007, (US$m): Rosetta Genomics Ltd
- Table 5.24: Financial details, year ending Dec 2007, (US$m):RXi Pharmaceuticals Corp
- Table 5.25: Financial details, year ending Jun 2007, (US$m): Senesco Technologies Inc
- Table 5.26: Financial details, year ending Dec 2007, (US$m): Senetek plc
- Table 5.27: Financial details, year ending Dec 2007, (m): Silence Therapeutics plc
- Table 5.28: Financial details, year ending Dec 2007, (US$m): Targeted Genetics Corp
- Table 5.29: Financial details, year ending Dec 2007, (Can$m): Tekmira Pharmaceuticals Corp
- Table 6.30: Common RNAi/Antisense Targets
- Table 6.31: Common RNAi/Antisense Targets (Contd.)
- Table 6.32: Targets of Antisense Oligonucleotides but not RNAis
- Table 6.33: WIPO RNAi Patents: Filing and Publication Dates
- Table 6.34: USPTO RNAi Patents: Filing and Publication Dates
- Table 6.35: WIPO RNAi Patents: Top Assignees
- Table 6.36: WIPO RNAi Patents: Top Assignees (Contd.)
- Table 6.37: WIPO RNAi Patents: Top Assignees (Contd.)
- Table 6.38: USPTO RNAi Patents: Top Assignees
- Table 6.39: Leading RNAi Oligonucleotide Originators
- Table 6.40: Key data: Forecasts for wet AMD, RSV therapy and acute renal failure ($m), 2012
