RNA Interference Markets

Since its discovery, the naturally occurring RNA (ribonucleic acid) interference effect has been acclaimed as the most exciting technical breakthrough in biological research in the last decade. Some industry analysts predict that RNA interference (RNAi) may even surpass PCR as a top technology. RNAi allows scientists to silence the expression or effect of a gene under study. This is known as gene knockdown. This field has rapidly emerged as a fast-growing new market. The purpose of this TriMark Publications report is to review the market for RNAi testing equipment and supplies.

RNAi is a mechanism in molecular biology where the presence of certain fragments of double-stranded RNA (dsRNA) interferes with the expression of a particular gene which shares a similar sequence with the dsRNA.

This study defines the dollar volume of sales, both worldwide and in the U.S., and analyzes the factors that influence market size and growth for RNAi testing. The main objectives of this study are to:

1) understand the different sectors of RNAi testing market and to look at a description of the instruments, reagents and supplies marketed by major companies in each segment;

2) obtain a complete understanding of the individual RNAi-testing platforms—from basic principles to clinical applications;

3) discover feasible market opportunities by identifying high-growth applications in different analytical diagnostic areas, with a focus on the biggest and expanding markets;

4) focus on global industry developments and trends through an in-depth analysis of the major world markets for RNAi measurement technology, including growth forecasts; and

5) present market figures related to the current value of RNAi testing, market projections, market share, key players and sector growth rates.  
 

1 Overview

1.1 Objectives

1.2 Scope

1.3 Methodology

1.4 Executive Summary

2 RNA Interference (RNAi)

2.1 Introduction

2.2 Overview of RNA Interference

2.2.1 Classes of Endogenous Small RNAs: siRNA, rasiRNA and miRNA

2.2.2 Mechanism of RNAi In Vivo

2.3 Glossary

2.4 What Areas of Research Does RNAi Impact?

2.4.1 RNAi Technology in Life Sciences Research, Drug Discovery and Development

2.4.2 RNAi Technology in Agriculture

3 Technology Trends in RNAi Space

3.1 Overview

3.2 End-User Segmentation

3.3 Technologies for Inducing RNAi

3.3.1 Methods for Detecting Gene Silencing

3.3.2 Comparison of Strategies for Target Validation

3.3.3 Libraries of siRNAs/shRNAs to Knock Down Genes En Masse

3.4 miRNA-Based RNAi: The Leading Edge of RNAi Space

3.4.1 Biological Function of miRNAs

3.4.2 Role of miRNAs in Disease

3.4.3 miRNA-based Diagnostics

3.4.4 miRNA-based Therapeutics

3.5 Market Shifts in RNAi Space: Where Is It Headed?

3.5.1 Intellectual Property and Patent Issues

3.5.2 Patents Covering the Molecular Characteristics of the RNAi Agent

3.5.3 Funding in RNAi Space: Grants, Venture Capital and IPOs

3.5.4 Partnerships, Alliances, Mergers and Acquisitions

3.6 SWOT Analysis of Technologies and Vendors in RNAi Space

4 RNAi Market Analysis

4.1 Overview

4.2 Market Data Collection and Respondent Pool

4.2.1 Respondent Pool Characteristics

4.3 RNAi Technologies / Products in Research Applications

4.4 RNAi Technologies/Products: Market Shares (Quantitative)

4.5 Quantitative Metrics of RNAi Usage: Market Opportunity and Growth

4.5.1 Growth of Various Segments in RNAi Space

4.5.2 Product Formats and Representation in RNAi Marketplace

4.6 RNAi Marketplace: Challenges, Unmet Needs and Drivers

4.6.1 Unmet Needs in RNAi Space

4.6.2 RNAi Market: Qualitative Growth Drivers

5 The RNAi Landscape

5.1 Overview

5.2 Market Segmentation of RNAi and Segment Characteristics

5.3 Quantitative Market Opportunities in RNAi Space

5.3.1 Opportunities in miRNA Space

5.3.2 Opportunities in siRNA/shRNA Space

5.4 RNAi Product Offerings and Associated Business Models

5.5 Challenges for RNAi Therapeutic Development

6 RNAi-Based Therapeutics: The Emerging Industry Landscape

6.1 Factors Contributing to the Success of RNAi Therapeutics

6.2 Advantages and Disadvantages of siRNA-based Drugs

6.3 Opportunities and Challenges with RNAi-based Therapeutics

6.4 The Gene Therapy Precedent

6.5 The Antisense Precedent

6.6 Interferon Response

6.7 Delivery of RNAi-based Therapeutics

6.8 Off-Target Effects

6.9 Overwhelming the Endogenous RNAi System—Affecting the microRNA Pathway

6.10 RNAi-based Therapeutics

6.10.1 Cancer

6.10.2 Cardiac Disease

6.10.3 Immunologic Disease

6.10.4 Infectious Disease

6.10.5 Inflammation

6.10.6 Lifestyle Therapeutics

6.10.7 Metabolic Disease

6.10.8 Neurologic Disease

6.10.9 Ophthalmic Disease

6.10.10 Renal Disease

6.10.11 Respiratory Disease

7 Company Profiles—U.S.

7.1 Alfacell Corporation

7.2 Allele Biotechnology and Pharmaceuticals, Inc.

7.3 Alnylam Pharmaceuticals

7.4 Ambion

7.5 Asuragen, Inc.

7.6 Avalon Pharmaceuticals, Inc.

7.7 B-Bridge International, Inc.

7.8 Bio-Rad Laboratories

7.9 Calando Pharmaceuticals, Inc.

7.10 Cepheid

7.11 Cequent

7.12 Clontech Laboratories, Inc.

7.13 CombiMatrix Corporation

7.14 Cyntellect, Inc.

7.15 CytRx Corp.

7.16 Dharmacon

7.17 Dicerna

7.18 Galenea Corporation

7.19 GeneCopoeia, Inc.

7.20 GeneThera, Inc.

7.21 Genlantis

7.22 GenoSensor

7.23 GRL, Inc.

7.24 IDT

7.25 Imgenex Corporation

7.26 Ingenuity Systems

7.27 Intradigm Corporation

7.28 Invitrogen

7.29 InvivoGen

7.30 Isis Pharmaceuticals, LLC

7.31 LC Sciences

7.32 Lentigen Corporation

7.33 Merck & Co., Inc.

7.34 Mirus Bio Corporation (Acquired by Roche)

7.35 Monsanto

7.36 Nastech Pharmaceutical Company, Inc.

7.37 New England BioLabs

7.38 Nucleonics, Inc.

7.39 Open Biosystems, Inc.

7.40 OPKO Health, Inc.

7.41 OriGene

7.42 Panomics, Inc.

7.43 PhaseRx

7.44 Promega Corp.

7.45 Quark Pharmaceuticals, Inc.

7.46 RXi Pharmaceuticals Corporation

7.47 Senetek PLC

7.48 Sigma-Aldrich

7.49 Sirna Therapeutics

7.50 Sirnaomics, Inc.

7.51 SomaGenics, Inc.

7.52 System Biosciences

7.53 Tacere Therapeutics

7.54 Targeted Genetics Corporation

7.55 Third Wave Technologies

7.56 Traversa

8 Company Profiles—Europe

8.1 Actigenics SA

8.2 Amaxa

8.3 AstraZeneca PLC

8.4 Cenix Bioscience GmbH

8.5 deVGen N.V.

8.6 DNAVision

8.7 Exiqon

8.8 Genovis

8.9 genOway

8.10 imaGenes GmbH

8.11 MWG Biotech AG

8.12 OZ Biosciences

8.13 Prosensa Holding

8.14 QIAGEN

8.15 RNAx GmbH (Germany)

8.16 Roche

8.17 Rosetta Genomics, Ltd.

8.18 Santaris Pharma A/S

8.19 Silence Therapeutics PLC

8.20 TaconicArtemis GmbH

8.21 TRANSAT

9 Company Profiles—Asia-Pacific

9.1 alphaGEN Co., Ltd.

9.2 Benitec, Ltd.

9.3 Bioneer

9.4 CytoPathfinder, Inc.

9.5 Genesis Research & Development Corp.

9.6 GeneDesign, Inc.

9.7 GNI Pharmaceutical Corporation

9.8 Koken Co., Ltd.

9.9 NanoCarrier Co., Ltd.

9.10 Oncolys Biopharma, Inc.

9.11 RealGene Bio-Technologies, Inc.

9.12 Samchully Pharmaceuticals

9.13 Samyang Corp.

9.14 Shanghai Biochip

9.15 Shanghai GenePharma Co.

9.16 Shanghai Genomics, Inc.

9.17 Transgene Biotek, Ltd.

10 Company Profiles—Rest of the World

10.1 Benitec, Ltd.

10.2 Tekmira

INDEX OF TABLES

Table 2.1: Advantages/Disadvantages of siRNAs and shRNAs as Inducers of RNAi in

Mammalian Cells

Table 2.2: Prevalence of siRNA-mediated RNAi in Disease Models, as Reported in

Scientific Literature

Table 2.3: RNAi-based Therapeutics in Clinical Trials

Table 2.4: Suppliers of Enabling Technologies and Tools in RNAi Space

Table 2.5: Suppliers of siRNA/shRNA Products (by Product/Service Class)

Table 3.1: Currently-Utilized Technologies for Inducing RNAi-Mediated Knockdown

Table 3.2: Comparison of mRNA Detection Technologies

Table 3.3: Strategies for Target Validation by Pharma/Biotech: RNAi and Others

Table 3.4: Comparison of Formats for Library-Based Gene Knockdown

Table 3.5: Characteristics of Various Publicly-Available Mouse and Human RNAi

Libraries

Table 3.6: Disease-Related miRNAs

Table 3.7: Publicly-Available Computational Programs for Identifying miRNA

Sequences/Targets

Table 3.8: Seminal Patents in RNAi

Table 3.9: Venture Capital Raised by Selected Companies in the RNAi Space

Table 3.10: List of Public Biotech Companies in RNAi Space

Table 3.11: SWOT Analysis of siRNA Oligonucleotides

Table 3.12: Transfection Reagents (Lipid-Mediated Delivery)

Table 3.13: shRNA Plasmid Constructs

Table 3.14: shRNA Pooled Libraries or Arrayed Collections (Retroviral/Lentiviral

Delivery)

Table 5.1: RNAi Market Segmentation and Research Settings

Table 5.2: Segment 1: Basic Life Science Research Utilizing RNAi Tools and

Technologies

Table 5.3: Segment 2: RNAi for Drug Discovery and Development

Table 5.4: Segment 3: RNAi Research for Development of RNAi Therapeutics

Table 5.5: Segment 4: RNAi Fee-for-Service Business

Table 5.6: Grants Awarded and Research Spending in miRNA Space, 2005 to 2008

Table 5.7: RNAi Opportunity and Market Size: Quantitative Model, 2006 to 2009

Table 5.8: Challenges for RNAi Therapeutic Development

Table 5.9: Technology Platforms used in RNAi Research

Table 6.1 : Similarities Between Antisense Oligonucleotides and siRNAs

Table 6.2: Disadvantages of siRNA Versus Antisense Oligonucleotides

Table 6.3: Delivery Vectors for siRNA and Hairpin-Encoding DNA for In Vivo

Experiments.

Table 6.4: Delivery Systems for siRNA and Hairpin-Encoding DNA for In Vivo

Experiments.

Table 6.5: Delivery Systems for RNAi-based Therapeutics

Table 6.6: Diseases for RNAi Therapeutics

Table 6.7: RNAi-Based Therapeutics Pipeline—Broken Out by Disease/Therapeutic

Area

Table 6.8: RNAi-Based Therapeutics Pipeline—Broken Out by Stage of Development

Table 6.9: Most Common Types of Cancer

Table 6.10: RNAi Therapeutics for Cancer in Phase I

Table 6.11: RNAi Therapeutics for Cancer in IND

Table 6.12: RNAi Therapeutics for Cardiac Disease in IND

Table 6.13: RNAi Therapeutics for Cardiac Disease in Pre-clinical/Research

Table 6.14: RNAi Therapeutics for Immunologic Disease in Pre-clinical / Research

Table 6.15: RNAi Therapeutics for Infectious Disease in Phase II

Table 6.16: RNAi Therapeutics for Infectious Disease in Phase I

Table 6.17: RNAi Therapeutics for Infectious Disease in IND

Table 6.18: RNAi Therapeutics for Infectious Disease in Pre-clinical/Research

Table 6.19: RNAi Therapeutics for Inflammatory Disease in Pre-clinical/Research

Table 6.20: RNAi Therapeutics for Life Style Therapies in IND

Table 6.21: RNAi Therapeutics for Metabolic Disease in Pre-clinical/Research

Table 6.22: RNAi Therapeutics for Neurologic Disease in Pre-clinical/Research

Table 6.23: RNAi Therapeutics for Ophthalmic Disease in Phase III

Table 6.24: RNAi Therapeutics for Ophthalmic Disease in Phase II

Table 6.25: RNAi Therapeutics for Ophthalmic Disease in Phase I

Table 6.26: RNAi Therapeutics for Ophthalmic Disease in Pre-clinical/Research

Table 6.27: RNAi Therapeutics for Renal Disease in Phase I

Table 6.28: RNAi Therapeutics for Respiratory Disease in Pre-clinical/Research

Table 10.1: Nucleonics Products and Programs Ongoing/Pipeline

Table 10.2: Quark’s Product Pipeline

Table 10.3: Santaris’ Product Pipeline

Table 10.4: Silence Therapeutics’ Product Pipeline

INDEX OF FIGURES

Figure 2.1: The miRNA Processing Pathway

Figure 2.2: Growth of Scientific Publications Addressing miRNAs, 2001 to 2008

Figure 2.3: Mechanisms of Small RNA-induced Gene Regulation

Figure 2.4: Types of RNAi Compounds

Figure 2.5: Breakdown of Scientific Publications Related to RNAi by Geographic

Region, 2007 to 2008

Figure 3.1: End-User Segmentation of RNAi Space

Figure 3.2: RNAi Patents Filed Globally, by Geographical Origin

Figure 3.3: Selected Deals in RNAi Space

Figure 4.1: Geographical Breakdown of RNAi End-User Survey Respondents

Figure 4.2: Affiliation of RNAi End-User Survey Respondents

Figure 4.3: Utilization of RNAi Technologies/Products by Respondent Pool

Figure 4.4: RNAi Utilization Period: Breakdown of Respondent Pool

Figure 4.5: Research Applications Using RNAi Technologies/Products

Figure 4.6: Types of RNAi Technologies Currently Utilized

Figure 4.7: RNAi Technologies Most Commonly Utilized Currently by End-Users

Figure 4.8: Types of RNAi Technologies: Expected Use in Six to 18 Months

Figure 4.9: Evolution of Market Shares of RNAi-Inducing Technologies

Figure 4.10: Number of Experiments Involving RNAi Conducted per Week

Figure 4.11: Range of Research Dollars Spent Monthly on RNAi Research

Figure 4.12: RNAi Experiment Throughput Correlated with Type of RNAi Technology

Used

Figure 4.13: Current RNAi Market Share by Segments

Figure 4.14: Growth/Decline Rate of RNAi Marketplace Segments (Six to 18 Months)

Figure 4.15: Product Formats Utilization for RNAi Research (Current and Six to

18 Months)

Figure 4.16: Companies Offering Product Formats for Currently-Used RNAi Products

Figure 4.17: RNAi Product Offerings/Formats Use (Projections—Six to 18 Months)

Figure 4.18: Percentage Change of RNAi Product Offerings/Formats (Six to 18

Months)

Figure 4.19: Key Challenges Faced by End-Users in RNAi Space

Figure 4.20: Unmet Needs in RNAi Space Ranked by Importance to End-Users

Figure 5.1: Growth and Evolution of miRNA Space

Figure 5.2: Elements of RNAi Value Chain

Figure 5.3: Growth and Evolution of RNAi (siRNA/shRNA) Space

Figure 5.4: Market Revenues Based on Components of siRNA Experimental Paradigm