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Outlook For Rnai, 2007: Sirna And Mirna In Biology, Diagnostics And Therapeutics

Business Insights
June 1, 2007
244 Pages - SKU: RET1526864

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Report overview

RNAi-based screens have provided new opportunities for the discovery and validation of novel therapeutic targets in areas such as cancer and infectious diseases. Since 2005, enormous strides have also been made in the development of RNAi-based therapeutic products, bringing them significantly closer to the market.

‘Outlook for RNAi 2007’ is a new report which provides a detailed insight into the most effective use of this novel technology in the drug discovery and development process. It puts into context where RNAi technology is being used as a tool of choice within academia and the pharma industry for functional genomic studies, drug target discovery and mechanism of action studies. The development status RNAi based therapeutics is covered in detail, highlighting the key companies in the field, outlining their pipelines and describing the results of completed clinical trials.

Understand how RNAi technology is being used to improve drug attrition rates and develop new therapeutic agents, and ensure that you are up-to-date with developments in this advancing field.

Key findings

The RNAi market is a relatively immature with global sales of approximately $447 million in 2006. It is projected to reach $0.9 billion by 2010, a compound annual growth rate (CAGR) of 19% between 2006-2010.
One of the main reasons for RNAi’s popularity stems from its utility as a molecular biology tool enabling the in vivo functional analysis of thousands of genes. Recent advances in the field include the design of new libraries of RNAi effectors, delivery systems and read-out methods.
In 2005, delivering RNAi triggers was the biggest obstacle in creating effective RNAi-based therapies. Subsequently, research into new and effective delivery methods has taken place at a frantic pace, although there are still major issues to be addressed. The first human trials of a systemic RNAi-based therapeutic were initiated in 2007 by Quark Biotech.
In the last two years, a more thorough understanding of the mechanism of action and intracellular pathways of miRNA has developed. miRNA is now an alternative gene knock-down technology that is being applied in research and for therapeutic and diagnostic applications.

Key questions answered

What will the growth of the RNAi market be over the next 5 years?
Who are the leading players in the RNAi market?
How are big pharma companies capitalizing on this technology through strategic alliances with specialized players?
Which areas of therapeutic interest are being explored with RNAi-based agents?
What are the Intellectual Property implications of RNAi research?
What challenges must be overcome for RNAi technology to yield commercially viable therapeutics and make a substantial improvement to drug-attrition rates?

Key issues examined in this report

The value and breakdown of sub-sector performances of the RNAi market between 2006-2015.
The therapy areas, progress of clinical research, potential drug sales and patent position of companies at the forefront of RNAi-based therapeutics.
The relative strengths and weaknesses of different approaches to RNAi delivery and selectivity.
The key trends that are currently affecting the RNAi market such as the development of new reagents, availability of custom synthesized siRNA, development of RNA microarrays and their impact on the R&D process
The development of miRNA for diagnostic and therapeutic purposes.

Outlook for RNAi: Executive Summary; Introduction; Design and Production of RNAi effectors; RNAi Research and Applications; Delivering RNAi therapeutics; RNAi Therapeutics: Progress and the Future; miRNA: diagnostics and therapeutics; Regulatory issues and patents; RNAi markets and trends
Chapter 1 Introduction: Summary; Introduction; DNA to RNA to protein - a primer; Antisense technologies; RNA interference; Overview and significance; RNAi’s mechanism; Post-transcriptional gene silencing (PGTS) by siRNA; miRNA pathways; Transcriptional gene silencing (TGS) by siRNA; Other non-coding RNAs; Advantages and disadvantages of RNAi; Report Outline
Chapter 2 Design and Production of RNAi: effectors; Summary; Introduction; Design of RNAi effectors; siRNA design algorithms; Building silencing efficiency; Avoiding off-target effects; Chemical modification; Avoiding immunostimulation; Finding the right target sites; miRNA mimics; Production of RNAi effectors; Chemical Synthesis; siRNA pools; Dicer-substrate siRNAs (DsiRNAs); Expression vectors; Plasmid expression vectors; Expression from a PCR product; Viral expression vectors; Conclusions
Chapter 3 RNAi Research and Applications: Summary; Introduction; Elucidating gene function; High throughput loss-of-function screening with RNAi; Choice of species and cell-line; RNAi reagents; Delivery method; Screening paradigm and format; Read-out methods; Building pathways; Investigating gene function in vivo; Applications of RNAi in drug discovery and development; Target discovery and validation; Optimization of drug therapy and development of personalized; medicine; miRNA Research; miRNA Databases and Algorithms; Identifying miRNAs, their targets and function; Isolation and enrichment of miRNA; Detection and quantification of miRNA; Functional analysis; Conclusions
Chapter 4 Delivering RNAi therapeutics: Summary; Introduction; Direct organ delivery; Stabilization of siRNAs; Non-viral delivery methods; Cholesterol and peptide conjugation; Liposomal delivery; Conjugation with cell penetrating peptides; Atelocollagen; Targeted polymeric delivery; Aptamer-siRNA complexes; FAb-siRNA complexes; MAb-siRNA complexes; Targeted nanoparticle delivery methods; Targeting with transferrin; Targeting with apatmers; Targeting with RGD peptide; Delivering RNAi via Plasmid DNA; Virosomes; Viral delivery methods; Conclusions
Chapter 5 RNAi Therapeutics: Progress and: the Future; Summary; Introduction; siRNA Therapeutics: Analysis by Therapeutic Area; Ocular Diseases; Age-related Macular Degeneration (AMD); Key RNAi players; Diabetic Retinopathy; Viral diseases; Respiratory Syncytial Virus (RSV); Key RNAi Players; HIV; Key RNAi players; Hepatitis C Virus (HCV); Key RNAi players; Hepatitis B Virus (HBV); Key RNAi players; Pandemic Flu; Key RNAi players; SARS; Key RNAi players; Herpes Simplex Virus (HSV); Other viral diseases; Respiratory diseases; Asthma and Chronic Obstructive Pulmonary Disease (COPD); Key RNAi Players; Cystic fibrosis (CF); Key RNAi players; Neurological diseases; Oncology; Angiogenesis; Key RNAi players; Oncogenes; Key RNAi players; Cardiovascular disease; Key RNAi players; Metabolic disorders; Diabetes and Obesity; Key RNAi players; Dermatology; Hair Removal; Key RNAi players; Pachyonychia congenita and related disorders; Key RNAi Players; Acute renal failure; Key RNAi players; Acute hearing loss/ pressure sores ototoxicity; Key RNAi players; Inflammatory Diseases; Key RNAi players; Conclusions
Chapter 6 miRNA: diagnostics and: therapeutics; Summary; Introduction; miRNA as a diagnostic tool; Companies developing miRNA-based diagnostics; Rosetta Genomics; Cepheid; Stratagene; miRNA-based therapeutics; Companies developing miRNA-based therapeutics; Alnylam Pharmaceuticals; Asuragen Inc; Santaris; Sirna; Conclusions
Chapter 7 Regulatory issues and patents: Summary; Introduction; Patents in RNAi; Seminal patents in RNAi; Alnylam; Sirna; Chemical modifications; DNA directed RNAi; Patents for specific RNAi targets; The future - more patent litigation?; MicroRNA; Tuschl III patents; Zamore and other patents; Regulatory considerations for RNAi therapies; Conclusions
Chapter 8 RNAi markets and trends: Summary; Introduction; The RNAi market; Market size and future trends; RNAi suppliers: synthesis and reagents; Alliances with big pharma for RNAi in R&D; Delivery of RNAi therapeutics; RNAi-based therapeutics; Big pharma alliances for RNAi-based therapeutics; miRNA-based diagnostics; Conclusion
List of Figures: Figure 1.1: Summary of antisense technologies other than RNAi; Figure 1.2: Timeline of RNAi discoveries; Figure 1.3: Significance of RNAi; Figure 1.4: Mechanism of post-transcriptional gene silencing by siRNA; Figure 1.5: Mechanism of miRNA mediated translational repression; Figure 2.1: Types of RNAi effector; Figure 2.2: Points of the RNAi pathway involved in determining silencing efficiency; Figure 2.3: Influence of guide RNA structure on siRNA efficiency; Figure 2.4: siRNA design that avoids off-target effects; Figure 2.5: Mechansims of immunostimulation with siRNA; Figure 2.6: Advantages and disadvantages of siRNA synthesis methods; Figure 2.7: Mechanism of ddRNAi compared to RNAi triggered by an siRNA; Figure 2.8: Nucleonics multi-targeting Anti-HBV drug; Figure 3.1: Considerations for a HT RNAi screen; Figure 3.2: Structures of silencing reagents for RNAi screening; Figure 3.3: Read-out methods for high throughput screens; Figure 3.4: Method of ArteMiceTM RNAi development; Figure 3.5: Peer reviewed miRNA publications (2001-2006); Figure 3.6: Comparison of Mirus’ LabelIT and enzymatically labelled samples; Figure 3.7: Inser Comparison of effectiveness of different knockdown methods t figuretitlehere, with date where appropriate; Figure 4.1: Chemical modifications of siRNAs increase stability and PK; Figure 4.2: Ribo-T siRNAs from Nastech Pharmaceuticals; Figure 4.3: Structure of a SNALP; Figure 4.4: Mirus Bio’s Dynamic PolyConjugates??delivery system for siRNA; Figure 4.5: Aptamer complexes for cell-type specific delivery of siRNAs; Figure 4.6: Heavy-chain antibody fragment for delivery of siRNAs to cell surface receptors; Figure 4.7: Targeted nanoparticles delivering siRNA; Figure 4.8: Aptamer directed nanoparticle formulation of docetaxel; Figure 4.9: Preparation of virosomes encapsulating siRNA; Figure 4.10: Viral delivery of shRNA; Figure 5.1: Development of AMD; Figure 5.2: siRNA targeting VEGF reduces blood vessel growth in the cornea; Figure 5.3: Visual Acuity data from Phase 1 studies of Sirna-027; Figure 5.4: Possible targets for suppressing HIV replication; Figure 5.5: siRNA mediated knockdown in Sirna’s primate model of HCV chimeric infection; Figure 5.6: Inhibition of three regions of the HCV genome by TT-033 for more than 2 months after a single administration to mice; Figure 5.7: Down regulation of Hepatitis B Surface Antigen after intravenous delivery of Nucleonic’s HBV candidate; Figure 6.1: Rosetta Genomics’ method for miRNA identification and validation; Figure 6.2: Illustration of Exiqon’s miRNA diagnostics; Figure 6.3: Structure of an antagomir; Figure 6.4: Asuragen’s development of miRNA-based therapeutics; Figure 6.5: let-7: tumor suppressor and future therapeutic; Figure 8.1: RNAi market sectors; Figure 8.2: Growth in the RNAi market 2006-2015
List of Tables: Table 1.1: Failures of antisense drugs; Table 1.2: Highly generalized comparison of siRNAs and miRNAs; Table 1.3: Advantages of RNAi; Table 1.4: Disadvantages of RNAi; Table 2.1: Selection of publicly available siRNA design tools; Table 2.2: Commercial siRNA design services; Table 2.3: Suppliers of custom and predesigned siRNAs; Table 2.4: Suppliers of siRNA pools and kits to create them; Table 2.5: Comparison of plasmid-based vectors and synthetic siRNA for research purposes; Table 2.6: Selection of plasmid vector products for siRNA generation; Table 2.7: Suppliers of kits for generating siRNA expression cassettes by PCR; Table 2.8: Advantages and disadvantages of different viral and non-viral expression vectors; Table 2.9: Selection of suppliers of viral vectors for gene silencing; Table 3.1: Suppliers of siRNA libraries for HT screening (3.1); Table 3.2: Commercial availability and coverage of shRNA libraries; Table 3.3: Summary of the pros and cons of the different silencing reagents for RNAi screening; Table 3.4: Advantages and disadvantages of RNAi knock-down in vivo; Table 3.5: Key databases and algorithms for miRNA target prediction; Table 3.6: Suppliers of miRNA mimics and inhibitors; Table 4.1: Non-viral methods for siRNA delivery; Table 5.1: RNAi therapeutics in, or close to, clinical development; Table 5.2: RNAi therapeutic targets in neurological disease; Table 5.3: Atugen’s pipeline of anticancer therapeutics; Table 6.1: miRNA expression profiling in human cancers; Table 7.1: Seminal patents in RNAi; Table 7.2: Issues in Tuschl I vs Tuschl II; Table 7.3: Licensees of Alnylam’s RNAi patents; Table 7.4: Chemical modifications - Sirna’s patent portfolio; Table 7.5: Therapeutic targets for RNAi - Sirna’s patent portfolio; Table 8.1: Companies involved in RNAi technologies, A-B; Table 8.2: Companies involved in RNAi technologies, C-G; Table 8.3: Companies involved in RNAi technologies, I-O; Table 8.4: Companies involved in RNAi technologies, P-Z; Table 8.5: Sales forecasts for total RNAi market, 2006-2015; Table 8.6: Sales forecasts for RNAi suppliers: synthesis and reagents, 2006 - 2015; Table 8.7: Pharma’s alliances for RNAi in R&D; Table 8.8: Sales forecasts for delivery of RNAi therapeutics, 2006-2015; Table 8.9: Costs associated with RNAi therapeutic development; Table 8.10: Potential value of therapy areas targeted by RNAi therapeutics, 2005 & 2010; Table 8.11: Sales forecasts for RNAi therapeutic drugs launched 2010-2015; Table 8.12: Pharma’s alliances for RNAi in R&D

Outlook For Rnai, 2007: Sirna And Mirna In Biology, Diagnostics And Therapeutics

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