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Published by: Business Insights
Published: Sep. 1, 2008 - 253 Pages
Table of Contents
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
List of Tables
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
Abstract"The Future of RNAi Therapeutics: Drug pipelines and prospects
""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."
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