RNAi Drug Delivery: Technologies and Global Markets

Report Summary

This report will provide:

An overview as to how the RNAi (ribonucleic acid interference) technologies market is applied in therapeutics, research and development (functional genomics, metabolism), drug discovery and agriculture
Analysis of global market trends, with data from 2009, estimates for 2010, and projections of compound annual growth rates (CAGRs) through 2015
Discussion of how the markets for RNAi are difficult to define as no RNAi-based product is in clinical development yet, and that the major use of RNAi reagents is in research but partially overlaps that of drug discovery and therapeutic development
Technology profiles of companies developing RNAi and antisense drugs, including pipeline information for each company
Evaluations of pending patents. 

INTRODUCTION

STUDY GOALS AND OBJECTIVES

This BCC Research report, RNAI Drug Delivery: Technologies and Global Markets, provides an in-depth assessment of recent progress and future opportunities for RNA interference (RNAi) players in major therapeutic and drug delivery areas, including infectious diseases, cancer, inflammation/immune dysfunction, central nervous system (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 RNAi-based therapeutic companies are profiled. This report also provides forecasts for market growth to 2015 across RNAi market segments, reviews the latest advances in RNAi drug delivery technologies, and assesses the competitive landscape across RNAi therapeutic areas.

REASONS FOR DOING THE STUDY

Since its discovery in 1998 by two scientists, the naturally occurring RNA interference effect has been hailed as the hottest technical breakthrough in biological research. It may even surpass polymerase chain reaction (PCR) as a top technology. RNA interference allows scientists to silence the expression or effect of a gene being studied. Known simply as RNAi, this field has rapidly emerged as a fast-growing new market. This report assesses this emerging commercial market and maps the key RNAi therapeutic and drug delivery players on the market landscape. The initial companies produced reagents and kits for the biological research reagents market to support basic and applied research in academia, government, and industry. A number of companies decided to take the long road to develop therapeutics. Others have developed drug delivery approaches for emerging RNAi therapeutics. This report analyzes emerging RNAi molecules and delivery technologies and examines new molecular targets and pipeline drugs. It also evaluates competitors and collaborators in the developing RNAi technologies and examines the latest RNAi-related patenting trends.

SCOPE OF REPORT

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 RNAi-based developers are profiled. This report also provides forecasts for market growth to 2015 across major RNAi market segments. This report provides an in-depth assessment of recent progress and future opportunities for RNAi players in major therapeutic areas.

INTENDED AUDIENCE

This report is an exhaustive study on the worldwide RNAi therapeutic and drug delivery market, giving crucial statistics and analysis on the delivery technologies, market structure, market size, key drug segments, important trends in technology development, major opportunities, and market shares of key players and drugs. This report may prove crucial for the firms or investors seeking engagement in the concerned sector. 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 small interfering RNA (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.

METHODOLOGY

Both primary and secondary research methodologies, interviews with key players in the RNAi field were used in preparing this report. Secondary sources include books, newspapers, trade journals, white papers, industry portals, government agencies, trade associations, monitoring industry news and developments, and through access to paid databases. The base year of the report is 2009, with forecast data provided through 2015. Historical, base year and forecast data are provided for each market segment of this report. Growth rates are determined through a compilation of data, including past trends, future trends, demographics, incidence, mortality, products in research and development (R&D), and current product growth, etc.

Table of Contents

CHAPTER ONE: INTRODUCTION
STUDY GOALS AND OBJECTIVES
REASONS FOR DOING THE STUDY
SCOPE OF REPORT
INTENDED AUDIENCE
METHODOLOGY
RELATED BCC STUDIES
ANALYST CREDENTIALS
BCC ONLINE SERVICES
DISCLAIMER

CHAPTER TWO: SUMMARY
SUMMARY
SUMMARY TABLE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, THROUGH 2015 ($ MILLIONS)
SUMMARY FIGURE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, 2009-2015 ($ MILLIONS)

CHAPTER THREE: OVERVIEW
OVERVIEW
THE PROMISE OF RNAI

TABLE 1 VARIOUS TECHNOLOGIES FOR GENE SUPPRESSION

RNAI TECHNOLOGY
RNAI MECHANISM: -HOW DOES IT WORK?
WHY SIRNA THERAPEUTICS?
SIRNA DELIVERY
RNAI DESIGN AND METHODS
Barriers to Delivery
Cellular Uptake and siRNAs
siRNA Delivery and Challenges

COMMERCIAL INTEREST
LIPOSOME-BASED DELIVERY
NONLIPOSOMAL METHODS
Nucleic-Acid Aptamers
Aptamers as Therapeutics

TABLE 2 THERAPEUTICALLY TARGETED APTAMERS

Aptamers for siRNA Delivery
Challenges-Aptamer-siRNA Delivery
Commercial Interest for Aptamers

ANTISENSE THERAPY
Selected Companies

CLASSES OF ENDOGENOUS SMALL RNAS
siRNA and rasiRNA
miRNA
OUTLOOK

CHAPTER FOUR: RNAI DRUG DELIVERY
RNAI DISCOVERED
IMPORTANCE OF DELIVERY FOR RNAI THERAPEUTICS
DRUG DELIVERY TECHNOLOGIES

TABLE 3 ROLE OF DRUG DELIVERY

NEED FOR NEW TECHNLOGIES
RNAI DELIVERY-TOOL FOR DRUG DISCOVERY AND DEVELOPMENT
siRNA Screening
shRNA Screening
CHALLENGES TO OVERCOME IN DELIVERING RNAI

TABLE 4 DIFFERENT RNAI DELIVERY APPROACHES

DIFFERENT DELIVERY TECHNIQUES
VECTOR-BASED DELIVERY
VECTOR-BASED RNAI FOR IN VIVO RNAI EXPERIMENTS

TABLE 5 COMPANIES WITH PLASMID RNAI VECTORS

Viral Vectors for Delivery of siRNA

TABLE 6 RNAI VIRAL VECTOR DELIVERY METHODS
TABLE 7 LENTIVIRAL DELIVERY PRODUCTS AND COMPANIES

NONVIRAL SIRNA DELIVERY
Hydrodynamic IV Injection
Electroporation

CARRIER-MEDIATED DELIVERY
Lipid-based siRNA Delivery
Polymer-Based siRNA Delivery
Combined Lipid and Polymer Carriers for a Better siRNA Delivery
Peptide-Mediated siRNA Delivery
Antibody-Mediated siRNA Delivery
Dendrimer-Mediated siRNA Delivery
Stem-cell-based Delivery
Light Controllable Delivery of siRNA

PROPRIETARY DELIVERY PLATFORMS
SYSTEMIC DELIVERY STRATEGIES

TABLE 8 THERAPEUTIC STRATEGIES

LOCALIZED DELIVERY
Targeted Cellular Delivery

EXPRESSION-BASED STRATEGIES
RNAI DELIVERY AND IMPACT ON THERAPEUTIC DEVELOPMENTS
New Developments
Tissue Delivery
RNAi as Drugs

CURRENT NONBIOLOGICAL DELIVERY VEHICLES FOR SIRNAS
Simple Excipients

TABLE 9 SIMPLE EXCIEPIENTS FOR RNAI DELIVERY

TRANSFECTION TECHNIQUES
Electroporation
Conjugates

TABLE 10 CONJUGATES, PEPTIDES, AND PROTEINS, OTHERS

Peptides and Proteins
Dynamic Polyconjugate Transfection
Polymer Carriers/Nanoparticles

TABLE 11 POLYMER CARRIERS/NANOPARTICLES FOR RNAI DELIVERY

Liposomes and Lipoplexes

TABLE 12 LIPOSOMES/LIPOPLEXES AND RNAI DELIVERY

Case Study-siRNA in Mammalian Cells
Gene Silencing (RNAi) in Neuronal Cells
Concerns about RNAi Delivery
KEY COMPANIES
siRNA Transfection

TABLE 13 COMPANIES INVOLVED IN DRUG DELIVERY TECHNOLOGIES FOR SIRNA

Delivery as Particles or Complexes
Delivery with Modifications
SUMMARY

CHAPTER FIVE: RNAI-BASED THERAPEUTICS
RNAI-BASED THERAPEUTICS: THE EMERGING INDUSTRY LANDSCAPE
DELIVERING RNAI THERAPEUTICS
RNAi and Cancer Therapeutics

REGULATORY CONCERNS
RNAI IN CLINICAL TRIALS
Clinical RNAi Pipeline

TABLE 14 THE GLOBAL RNA INTERFERENCE THERAPEUTIC PIPELINE

THE MOST ADVANCED RNAI CANDIDATE
Alnylam’s ALN-RSV: Respiratory Syncytial Virus
ALN-RSV01 Treatment of Respiratory Syncytial Virus Infection in Lung Transplant Patients

THERAPEUTIC RNAI-RECENT DEVELOPMENTS
Case Study-ALN-VSP02
SNALP Delivery
Other Candidates in Clinical Trials
Unexpected Problems and Possible Solutions for RNAi-Based Therapies

CHAPTER SIX: RNAI LANDSCAPE
FUTURE TRENDS
WHAT IS RNA INTERFERENCE?
Locked Nucleic Acid

RNAI AS A RESEARCH TOOL
THE IMPORTANCE OF RNAI DESIGN
FACTS AND TIMELINES
MARKET GROWTH
GLOBAL MARKET FOR RNAI
TABLE 15 GLOBAL RNAI RESEARCH SALES AND FORECAST, THROUGH 2015 ($ MILLIONS)

FIGURE 1 GLOBAL RNAI RESEARCH SALES, 2008-2015 ($ MILLIONS)
TABLE 16 GLOBAL TOTAL MARKET FOR RNAI, THROUGH 2015 ($ MILLIONS)
FIGURE 2 GLOBAL TOTAL MARKET FOR RNAI, 2009-2015 ($ MILLIONS)

RNAI TECHNOLOGIES/PRODUCTS IN RESEARCH APPLICATIONS
RNAi Screening for Drug Discovery
Individual versus Pooled siRNAs
Necessary Controls
High-Throughput siRNA Delivery

RNAI TOOL AND REAGENT MARKET
Transfection Products
Key Companies

TABLE 17 RNAI COMPANIES WITH TRANSFECTION REAGENTS

RNAi Reagents
Lipid Transfection
In Vivo Reagents and Therapeutics
RNAi Experiments in Animals

RNAI AND THERAPEUTICS

TABLE 18 IN VIVO DELIVERY

Viral-based Transfections

TABLE 19 VIRAL BASED DELIVERY TECHNOLOGIES

Other Methods
RNAi Reagents and Kits

TABLE 20 RNAI TOOL/REAGENT COMPANIES

KEY COMPANIES
Thermo Fisher
Invitrogen-Results of Operations

TABLE 21 INVITROGEN-REVENUES FOR THE FIRST 6 MONTHS, 2009-2010 (MILLIONS)

Lonza

TABLE 22 LONZA-REVENUES, 2009 ($ MILLIONS)

Lonza’s RNAi Products
THE FUTURE OF RNAI THERAPEUTICS
CHAPTER SEVEN: RNAI DRUG DELIVERY-MARKET DATA
MARKET SIZE AND GROWTH
DRUG DELIVERY TECHNOLGIES AND MARKET
GLOBAL PHARMACEUTICAL MARKET

TABLE 23 GLOBAL PHARMACEUTICAL SALES, THROUGH 2015 ($ BILLIONS)
FIGURE 3 GLOBAL PHARMACEUTICAL SALES, 2008-2015 ($ BILLIONS)

UNITED STATES PHARMACEUTICAL INDUSTRY
TOP 20 PHARMACEUTICAL COMPANIES IN 2009

TABLE 24 TOP 20 PHARMACEUTICAL COMPANIES, 2009 ($ MILLIONS: BASED ON 2008 SALES)
TABLE 25 TOP 20 PHARMACEUTICAL COMPANIES, 2010 ($ MILLIONS: BASED ON 2009 SALES))
TABLE 26 TWELVE LARGEST PHARMACEUTICAL COMPANIES, 2010 ($ MILLIONS)

DRUG DELIVERY MARKET
GLOBAL MARKET

TABLE 27 TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 4 TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
TABLE 28 GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 5 GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)

ORAL DRUG DELIVERY

TABLE 29 GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 6 GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
TABLE 30 GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)

PULMONARY DRUG DELIVERY

FIGURE 7 GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)

IMPLANTABLE AND INJECTABLE DRUG DELIVERY

TABLE 31 GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 8 GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, 2009-2015 ($ MILLIONS)

TRANSDERMAL DRUG DELIVERY

TABLE 32 GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 9 GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)

U.S. Market

TABLE 33 U.S. DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 10 U.S. DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)

RNAI MARKET ANALYSIS
RNAI NUCLEIC ACID DRUG DELIVERY MARKET

TABLE 34 GLOBAL RNAI DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 11 GLOBAL RNAI DRUG DELIVERY TECHNOLOGIES MARKET, 2007-2015 ($ MILLIONS)

Aptamers and RNAi Market

TABLE 35 GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 12 GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2015 ($ MILLIONS)

TARGETED DELIVERY
THE TOTAL NANOPARTICLE MARKET

TABLE 36 GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 13 GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
TABLE 37 GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 14 GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)

RNAI DELIVERY APPROACHES AND CURRENT DEVELOPMENTS
DEVELOPMENT OF RNAI-BASED THERAPEUTICS
Delivery of Therapeutics to the Target Tissues
RNAi Drugs under Investigation
Naked siRNA
Companies/Latest Developments
Lipid and Polymer-Based Systems

TABLE 38 LIPID-BASED TECHNOLOGIES-COMPANIES
TABLE 39 POLYMER-BASED AND LIPOSOMAL RNAI DELIVERY SYSTEMS (%)

RNAI INTO THE PATIENTS
Polyconjugate Technologies
Polymer-Based Systems—Limitations
Nanoparticles

TABLE 40 NANOPARTICLES TECHNOLOGIES-COMPANIES

Single siRNA Delivery

TABLE 41 SINGLE RNAI DELIVERY TECHNOLOGIES-COMPANIES

NEW DELIVERY TECHNOLOGIES
SYSTEMIC DELIVERY
SYSTEMIC DELIVERY STRATEGIES
Challenges with RNAi Drug Delivery
RNAI THERAPEUTIC MARKET

TABLE 42 SELECTED RNAI-BASED DRUGS IN DEVELOPMENT

COMMERCIAL POTENTIAL OF RNAI
RNAI’S THERAPEUTIC POTENTIAL
COMPANIES AND SIRNA
How Does RNAi Work?
RNAis and Preclinical Studies
CEQ508 in Clinic
RNAi as a Therapy

GLOBAL MARKET OF THERAPEUTIC RNAI
RNAi Therapeutic Market to Play a Key Role by 2020

TABLE 43 GLOBAL MARKET FOR THERAPEUTIC RNAI, THROUGH 2015 ($ MILLIONS)
FIGURE 15 GLOBAL MARKET FOR THERAPEUTIC RNAI, 2009-2015 ($ MILLIONS)
TABLE 44 RNAI THERAPEUTIC COMPANIES

MicroRNA and Market

TABLE 45 MICRORNA MARKET AND FORECAST, THROUGH 2015 ($ MILLIONS)
FIGURE 16 MICRORNA MARKET AND FORECAST, 2009-2015 ($ MILLIONS)
TABLE 46 TOTAL MIRNA MARKET SALES, 2008 (%)

RNAI LICENSING DEALS

TABLE 47 MAJOR RNAI LICENSING DEALS, 2005-2010

Recent Trends
New RNAi Delivery Technologies
TARGETED DELIVERY

TABLE 48 SYSTEMIC DELIVERY TECHNOLOGIES-COMPANIES

KEY THERAPEUTIC COMPANIES AND DEVELOPMENTS

TABLE 49 LEAD COMPANIES DEVELOPING RNAI-BASED DRUGS

ALNYLAM

TABLE 50 ALNYLAM PHARMACEUTICALS-NET REVENUE, 2009 AND 2010 ($ THOUSANDS)

Roche Alliance

TABLE 51 ALNYLAM’S TOTAL NET REVENUES FROM RESEARCH COLLABORATORS, 2009 AND 2010 ($ THOUSANDS)

Takeda Alliance
Other-Discovery and Development Alliances
Isis Collaboration
Novartis Broad Alliance
Product Alliances
Kyowa Hakko Kirin Alliance
Cubist Alliance
Government Funding
NIH Contract

TABLE 52 ALNYLAM’S DEVELOPMENT PIPELINE

TEKMIRA
Funding
Tekmira’s LNP Technology

TABLE 53 TEKMIRA’S DEVELOPMENT PIPELINE

Tekmira’s Pipeline
TKM-ApoB
TKM-PLK1
TKM-Ebola
Roche
Merck
Bristol-Myers Squibb
U.S. Army Medical Research Institute for Infectious Diseases (USAMRIID) Research Agreement
Takeda
Pfizer

TABLE 54 TEKMIRA’S PIPELINE

Revenue

TABLE 55 TEKIMIRA REVENUE ($ MILLIONS)

RXI PHARMACEUTICALS
Pipeline
TABLE 56 RXI PHARMACEUTICALS’ DEVELOPMENT PIPELINE
RXi Agreements
Philips
Other Collaborative Agreements
Scientific Achievements
NIH Grant
Financial Highlights
MARINA BIOTECH
Pipeline

TABLE 57 MARINA BIOTECH’S DEVELOPMENT PIPELINE

Revenue

TABLE 58 MARINA BIOTECH REVENUE, 2008 AND 2009 ($ THOUSANDS)
TABLE 59 MARINA BIOTECH REVENUE FROM CUSTOMERS AS A PERCENTAGE OF TOTAL REVENUE, 2008 AND 2009 (%)

Ongoing Collaborations
Beth Israel Deaconess Medical Center-Boston, Massachusetts
Institut Pasteur Paris, France
University of British Columbia (UBC)/Vancouver Prostate Centre (VPC)-Vancouver, British Columbia, Canada
University of Helsinki-Helsinki, Finland

TABLE 60 MARINA BIOTECH SELECTED COLLABORATIONS

CALANDO PHARMACEUTICALS
CALAA-01
ISIS PHARMACEUTICALS
Pipeline

TABLE 61 ISIS PHARMACEUTICALS DRUG DEVELOPMENT PIPELINE

Strategic Alliances
Pharmaceutical Partners

TABLE 62 ISIS PHARMACEUTICALS, REVENUE, 2009-2010 ($ THOUSANDS)

SILENCE THERAPEUTICS
Proprietary Delivery Technologies
Pipeline

TABLE 63 SILENCE THERAPEUTICS-DEVELOPMENT PIPELINE

Collaborations
AstraZeneca
Dainippon Sumitomo Pharma
Quark Pharmaceuticals
Financial Highlights

TABLE 64 SILENCE THERAPEUTICS-REVENUE, 2009 AND 2010 ($ MILLIONS)
QUARK PHARMACEUTICALS
Clinical Studies

TABLE 65 QUARK PHARMACEUTICALS CURRENT CLINICAL TRIALS

Pipeline

TABLE 66 QUARK PHARMACUETICALS-DEVELOPMENT PIPELINE

ROCHE
MERCK SERONO
SIRNAOMICS
ACCESS PHARMACEUTICALS
KEY MICRORNA THERAPEUTIC COMPANIES
MIRNA THERAPEUTICS
Pipeline

TABLE 67 MIRNA-DEVELOPMENT PIPELINE

REGULUS
Pipeline
Partnerships

LOCAL THERAPY
DRUG DELIVERY IN CANCER
RNAI MARKETPLACE CHALLENGES
RNAI’S GROWTH AND FUTURE

CHAPTER EIGHT: GLOBAL MARKET FOR RNAI TECHNOLOGY
FUTURE
RNAI- DISCOVERY AND GROWTH
RNAI THERAPEUTICS FORECAST
COMPANY PROFILES
THERAPEUTIC COMPANIES
Alnylam Pharmaceuticals
Dicerna Pharmaceuticals
RXi Pharmaceuticals
ProNAi Therapeutics
Somagenics

DELIVERY COMPANIES
Sirnaomics
Calondo Pharmaceuticals
Tacere Therapeutics
PhaseRx
Marina Biotech
Tekmira
Isis Pharmaceuticals
Silence Therapeutics
Quark Pharmaceuticals
Merck
Access Pharmaceuticals
AparnaBio
Traversa Therapeutics

MICRORNA COMPANIES
Regulus
Asuragen
Mirna Therapeutics

REAGENT/TOOL COMPANIES
Ambion
Invitrogen
B-Bridge International
Bio-Rad Laboratories
Clontech Laboratories
Cyntellect
Dharmacon
Integrated DNA Technologies (IDT)
Genecopoeia
GeneThera
Genlantis
Genosensor
General Research Laboratory
Imgenex
Invivogen
New England Biolabs
Open Biosystems
Origene Technologies
Panomics
Promega Corporation
Sigma-Aldrich
System Biosciences

COMPANY PROFILES-EUROPE
ACTIGENICS SA
AMAXA
Cenix Bioscience Gmbh
GENOVIS
PROSENSA HOLDING
QIAGEN GMBH
RNAX GMBH (GERMANY)
SANTARIS PHARMA A/S
COMPANY PROFILES-ASIA-PACIFIC
ALPHAGEN
BENITEC
BIONEER
CYTOPATHFINDER, INC.
GENESIS RESEARCH & DEVELOPMENT CORP
KOKEN CO. LTD.
ONCOLYS BIOPHARMA, INC.
SHANGHAI GENEPHARMA CO. LTD.
SHANGHAI GENOMICS
TRANSGENE BIOTEK

CHAPTER NINE: MARKET SHIFTS IN RNAI SPACE
MARKET SHIFTS IN RNAI SPACE
INTELLECTUAL PROPERTY AND PATENT ASPECTS

TABLE 68 MAJOR RNAI COMPANIES IN THE US-PATENT DISTRIBUTION

FUTURE DEVELOPMENTS
SOME SELECTED PATENTS
RECENT PATENT NEWS

TABLE 69 PATENTS

LICENSES FOR RNAI PATENTS
KEY COMPANIES AND PATENTS
ALNYLAM
RXI
ISIS
QUARK
BENITEC
PARTNERSHIPS, ALLIANCES, MERGERS, AND ACQUISITIONS
ALNYLAM COLLABORATORS: SIGNIFICANT NEW ADVANCES IN RNAI DELIVERY
Alnylam, Medtronic, and CHDI Foundation
Alnylam and Tekmira

RECENT FUNDING
Marina Biotech Announces Financing for up to $8.7 Million
Tekmira and Collaborators Awarded $2.4 Million NIH Grant for Development of RNAi Therapeutics to Treat Ebola and Marburg Viral Infections
RXi Pharmaceuticals Awarded $1 Million in IRS Grants to Advanced RNAi Therapeutics
Sanofi-Aventis Completes $10 Million Equity Investment in Regulus Therapeutics
Tekmira Provides Update on NASDAQ Listing

CONCLUSIONS
BIBLIOGRAPHY
BIBLIOGRAPHY

LIST OF TABLES

SUMMARY TABLE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, THROUGH 2015 ($ MILLIONS)
TABLE 1 VARIOUS TECHNOLOGIES FOR GENE SUPPRESSION
TABLE 2 THERAPEUTICALLY TARGETED APTAMERS
TABLE 3 ROLE OF DRUG DELIVERY
TABLE 4 DIFFERENT RNAI DELIVERY APPROACHES
TABLE 5 COMPANIES WITH PLASMID RNAI VECTORS
TABLE 6 RNAI VIRAL VECTOR DELIVERY METHODS
TABLE 7 LENTIVIRAL DELIVERY PRODUCTS AND COMPANIES
TABLE 8 THERAPEUTIC STRATEGIES
TABLE 9 SIMPLE EXCIEPIENTS FOR RNAI DELIVERY
TABLE 10 CONJUGATES, PEPTIDES, AND PROTEINS, OTHERS
TABLE 11 POLYMER CARRIERS/NANOPARTICLES FOR RNAI DELIVERY
TABLE 12 LIPOSOMES/LIPOPLEXES AND RNAI DELIVERY
TABLE 13 COMPANIES INVOLVED IN DRUG DELIVERY TECHNOLOGIES FOR SIRNA
TABLE 14 THE GLOBAL RNA INTERFERENCE THERAPEUTIC PIPELINE
TABLE 15 GLOBAL RNAI RESEARCH SALES AND FORECAST, THROUGH 2015 ($ MILLIONS)
TABLE 16 GLOBAL TOTAL MARKET FOR RNAI, THROUGH 2015 ($ MILLIONS)
TABLE 17 RNAI COMPANIES WITH TRANSFECTION REAGENTS
TABLE 18 IN VIVO DELIVERY
TABLE 19 VIRAL BASED DELIVERY TECHNOLOGIES
TABLE 20 RNAI TOOL/REAGENT COMPANIES
TABLE 21 INVITROGEN-REVENUES FOR THE FIRST 6 MONTHS, 2009-2010 (MILLIONS)
TABLE 22 LONZA-REVENUES, 2009 ($ MILLIONS)
TABLE 23 GLOBAL PHARMACEUTICAL SALES, THROUGH 2015 ($ BILLIONS)
TABLE 24 TOP 20 PHARMACEUTICAL COMPANIES, 2009 ($ MILLIONS: BASED ON 2008 SALES)
TABLE 25 TOP 20 PHARMACEUTICAL COMPANIES, 2010 ($ MILLIONS: BASED ON 2009 SALES))
TABLE 26 TWELVE LARGEST PHARMACEUTICAL COMPANIES, 2010 ($ MILLIONS)
TABLE 27 TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 28 GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 29 GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 30 GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 31 GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 32 GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 33 U.S. DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 34 GLOBAL RNAI DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 35 GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 36 GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 37 GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2015 ($ MILLIONS)
TABLE 38 LIPID-BASED TECHNOLOGIES-COMPANIES
TABLE 39 POLYMER-BASED AND LIPOSOMAL RNAI DELIVERY SYSTEMS (%)
TABLE 40 NANOPARTICLES TECHNOLOGIES-COMPANIES
TABLE 41 SINGLE RNAI DELIVERY TECHNOLOGIES-COMPANIES
TABLE 42 SELECTED RNAI-BASED DRUGS IN DEVELOPMENT
TABLE 43 GLOBAL MARKET FOR THERAPEUTIC RNAI, THROUGH 2015 ($ MILLIONS)
TABLE 44 RNAI THERAPEUTIC COMPANIES
TABLE 45 MICRORNA MARKET AND FORECAST, THROUGH 2015 ($ MILLIONS)
TABLE 46 TOTAL MIRNA MARKET SALES, 2008 (%)
TABLE 47 MAJOR RNAI LICENSING DEALS, 2005-2010
TABLE 48 SYSTEMIC DELIVERY TECHNOLOGIES-COMPANIES
TABLE 49 LEAD COMPANIES DEVELOPING RNAI-BASED DRUGS
TABLE 50 ALNYLAM PHARMACEUTICALS-NET REVENUE, 2009 AND 2010 ($ THOUSANDS)
TABLE 51 ALNYLAM’S TOTAL NET REVENUES FROM RESEARCH COLLABORATORS, 2009 AND 2010 ($ THOUSANDS)
TABLE 52 ALNYLAM’S DEVELOPMENT PIPELINE
TABLE 53 TEKMIRA’S DEVELOPMENT PIPELINE
TABLE 54 TEKMIRA’S PIPELINE
TABLE 55 TEKIMIRA REVENUE ($ MILLIONS)
TABLE 56 RXI PHARMACEUTICALS’ DEVELOPMENT PIPELINE
TABLE 57 MARINA BIOTECH’S DEVELOPMENT PIPELINE
TABLE 58 MARINA BIOTECH REVENUE, 2008 AND 2009 ($ THOUSANDS)
TABLE 59 MARINA BIOTECH REVENUE FROM CUSTOMERS AS A PERCENTAGE OF TOTAL REVENUE, 2008 AND 2009 (%)
TABLE 60 MARINA BIOTECH SELECTED COLLABORATIONS
TABLE 61 ISIS PHARMACEUTICALS DRUG DEVELOPMENT PIPELINE
TABLE 62 ISIS PHARMACEUTICALS, REVENUE, 2009-2010 ($ THOUSANDS)
TABLE 63 SILENCE THERAPEUTICS-DEVELOPMENT PIPELINE
TABLE 64 SILENCE THERAPEUTICS-REVENUE, 2009 AND 2010 ($ MILLIONS)
TABLE 65 QUARK PHARMACEUTICALS CURRENT CLINICAL TRIALS
TABLE 66 QUARK PHARMACUETICALS-DEVELOPMENT PIPELINE
TABLE 67 MIRNA-DEVELOPMENT PIPELINE
TABLE 68 MAJOR RNAI COMPANIES IN THE US-PATENT DISTRIBUTION
TABLE 69 PATENTS

LIST OF FIGURES

SUMMARY FIGURE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, 2009-2015 ($ MILLIONS)
FIGURE 1 GLOBAL RNAI RESEARCH SALES, 2008-2015 ($ MILLIONS)
FIGURE 2 GLOBAL TOTAL MARKET FOR RNAI, 2009-2015 ($ MILLIONS)
FIGURE 3 GLOBAL PHARMACEUTICAL SALES, 2008-2015 ($ BILLIONS)
FIGURE 4 TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 5 GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 6 GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 7 GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 8 GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, 2009-2015 ($ MILLIONS)
FIGURE 9 GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 10 U.S. DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 11 GLOBAL RNAI DRUG DELIVERY TECHNOLOGIES MARKET, 2007-2015 ($ MILLIONS)
FIGURE 12 GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2015 ($ MILLIONS)
FIGURE 13 GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 14 GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, 2009-2015 ($ MILLIONS)
FIGURE 15 GLOBAL MARKET FOR THERAPEUTIC RNAI, 2009-2015 ($ MILLIONS)
FIGURE 16 MICRORNA MARKET AND FORECAST, 2009-2015 ($ MILLIONS)