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Outlook for RNAi, 2007: siRNA and miRNA in Biology, Diagnostics and Therapeutics

Posted on: Tuesday, 28 August 2007, 06:13 CDT

Reportlinker.com announces that a new market research report related to the worldwide biotechnology industry is now available to its catalogue.

Outlook for RNAi, 2007: siRNA and miRNA in biology, diagnostics and therapeutics

http://www.reportlinker.com/p054680/Outlook-RNAi.html

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.

Table of content

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

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Source: Business Wire

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