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市場調査レポート

RNAiドラッグデリバリー:技術及び世界市場

RNAi Drug Delivery: Technologies and Global Markets

発行 BCC Research 商品コード 306915
出版日 ページ情報 英文 261 Pages
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RNAiドラッグデリバリー:技術及び世界市場 RNAi Drug Delivery: Technologies and Global Markets
出版日: 2014年06月30日 ページ情報: 英文 261 Pages
担当者のコメント
BCC Research社のレポートは、Reuter、Wall Street Journal、Fortuneなどで引用されることも多く、それぞれの業界の識者の意見として重きを置かれています。同社レポートを複数冊ご購入の場合年間パッケージのご契約がお得です。詳細は右の担当者のおすすめ商品よりご覧下さい。
概要

RNAi(RNA干渉)ドラッグデリバリーの世界市場規模は、2012年概算実績93億ドル、2013年の同117億ドルから、以降数年にわたって27.2%の複合年間成長率を示し、2018年にはおよそ388億ドルに達する見込み。

当レポートでは、RNAiドラッグデリバリーの世界市場に注目し、その概況および2013-2018年の期間における取引規模の推移を分析・予測するほか、技術や関連療法、市場動向、競合環境、主要企業のプロフィールなどの最新情報を集めています。

第1章 イントロダクション

  • 調査の目標と目的
  • 調査理由
  • レポートの範囲
  • 推奨利用者層
  • 調査方法
  • 関連BCC Research社レポート
  • アナリスト経歴
  • BCC Research社WEBサイト
  • 免責事項

第2章 サマリー

第3章 概要

  • 概要
    • RNAiとは
    • 療法としてのRNAi
  • RNAiの有望性
  • RNAi技術
    • RNAiのメカニズム:その作用
    • siRNA(小干渉RNA)療法を用いる理由
    • siRNAの送達
    • siRNAの設計と方法論
      • 送達の障害
      • 細胞取り込みとsiRNA
      • siRNA送達と問題点
  • 商業的利益
    • リポソーム利用による送達
    • 非リポソーム方式
    • アンチセンス療法
    • 内因性低分子RNAの等級
    • 将来見通し

第4章 RNAiドラッグデリバリー

  • RNAiの発見
  • RNAi療法における送達技術の重要性
    • ドラッグデリバリー技術
  • 新技術の必要性
  • ドラッグデリバリー・開発に向けてのsiRNA送達ツール
    • siRNAスクリーニング
    • shRNAスクリーニング
  • 克服すべきRNAi送達の問題点
  • さまざまな送達技法
    • ベクター利用の送達
    • 生体内実験に向けたベクター利用のRNAi送達
      • siRNA送達用ウィルスベクター
    • 非ウィルスsiRNA送達
    • 特許送達プラットフォーム
    • 全身送達策
    • 局所送達
    • 発現ベース策
    • RNAi送達と療法開発への影響
    • siRNAを対象とした現行の非生物学的送達媒体
    • トランスフェクション技法
    • 有力企業
    • サマリー

第5章 RNAi利用の療法

  • RNAi利用の療法:新たな産業環境
  • RNAi送達療法
    • RNAiとがん治療
  • 規制問題
    • 治験:RNAi
    • 臨床における他の候補策
    • 終了プログラム
    • 他のプログラム
  • 治療用RNAiの開発
    • ALN-VSP02
    • Alnylam社のALN-RSV:呼吸器合胞体(RS)ウイルス
    • 肺移植患者に対する呼吸器合胞体(RS)ウイルス感染のALN-RSV01治療
  • 課題:RNAi利用の療法

第6章 - RNAi環境

  • 今後の動向
    • RNA干渉とは
    • 研究手段としてのRNAi
    • RNAi設計の重要性
    • 事実と時間軸
    • 市場成長
    • RNAiの世界市場
    • 研究用のRNAi技術・製品
    • RNAiツールおよび試薬市場
    • RNAiと治療法
    • 有力企業
    • RNAi療法の将来性

第7章 RNAiドラッグデリバリー - 市場データ

  • 市場動向
  • 市場規模および成長率
    • RNAi薬剤
    • 医薬品の世界市場
  • ドラッグデリバリー技術環境
    • ドラッグデリバリー市場
    • 経肺ドラッグデリバリー
  • RNAi市場分析
    • 有力企業
    • RNAi市場動態
    • RNAi核酸ドラッグデリバリー
    • 核酸アプタマー系ナノ粒子
    • 標的送達
    • 総合ナノ粒子市場
    • 標的体組織に対する療法の送達
    • RNAi送達手法と現在の開発状況
    • RNAi利用療法の開発
    • ナノ粒子利用の送達法
    • リポソームとナノ粒子
    • ポリマー
    • CALANDO PHARMACEUTICALS
    • INTRADIGM
  • メソ多孔質シリカナノ粒子
  • たんぱく質またはペプチド系ナノ粒子
  • バクテリオファージ系ナノ粒子
    • SOLSTICE BIOLOGICS
    • GENZYME
    • QUARK PHARMACEUTICALS
  • 患者へのRNAi投与
    • ROCHE
  • DICERNA PHARMACEUTICALS
  • 結び

第8章 RNAiドラッグデリバリー策と現在の市場動態

  • 全身送達法
  • RNAiドラッグデリバリーの問題点
  • RNAi療法市場
  • RNAiの商業的発展性
  • RNAiの治療学的発展性
  • 近年の展開

第9章 企業プロフィール

第10章 RNAiに関わる市場の変化

  • 知的所有権と特許問題
  • 最新特許ニュース
  • RNAi特許ライセンス
  • 主要企業と特許
  • 提携、連合、事業買収
  • 結び

第11章 参考文献一覧

目次
Product Code: BIO076B

REPORT HIGHLIGHTS

The global market for RNAi drug delivery technologies reached nearly $9.3 billion in 2012 and nearly $11.7 billion in 2013. This market is expected to grow to $38.8 billion in 2018, with a compound annual growth rate(CAGR) of 27.2% from 2013 to 2018.

This report provides:

  • An overview of the global market for RNA interference (RNAi) in major therapeutic and drug delivery areas.
  • Analyses of global market trends, with data from 2012, estimates for 2013, and projections of compound annual growth rates (CAGRs) through 2018.
  • Details on application areas including infectious diseases, cancer, inflammation/immune dysfunction, central nervous system (CNS) disorders, and cardiology/metabolism.
  • Identification of the driving factors for demand in the market
  • Discussion of emerging technologies.
  • Comprehensive company profiles of major players in the industry.

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 2018 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 2018 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.

ANALYST'S CREDENTIALS

Usha Nagavarapu is an experienced pharmaceutical professional with business development experience. She has more than 10 years of preclinical, alliance management, discovery, and technology development marketing experience. Her strong focus areas include oncology and cardiovascular diseases, with expertise in molecular and cell biology and complex cell-based biological assays ranging from drug discovery, in vitro and in vivo screening, in vivo model development, and pharmacokinetics. She has experience working with early start-ups.

Table of Contents

CHAPTER 1 - INTRODUCTION

  • STUDY GOALS AND OBJECTIVES
  • REASONS FOR DOING THE STUDY
  • SCOPE OF REPORT
  • INTENDED AUDIENCE
  • METHODOLOGY
  • RELATED BCC RESEARCH REPORTS
  • ANALYST'S CREDENTIALS
  • BCC RESEARCH WEBSITE
  • DISCLAIMER

CHAPTER 2 - SUMMARY

  • SUMMARY TABLE: GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, THROUGH 2018 ($ MILLIONS)
  • SUMMARY FIGURE: GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, 2013-2018 ($ MILLIONS)

CHAPTER 3 - OVERVIEW

  • OVERVIEW
    • WHAT IS RNAI?
    • RNAI AS THERAPY
  • 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 METHODOLOGIES
      • 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
      • Nanotechnology-based Delivery
      • Nanoparticles
      • Advantages
        • TABLE 3: REQUIRED STEPS FOR DEVELOPING EFFECTIVE RNAI DRUG
    • ANTISENSE THERAPY
      • Selected Companies
    • CLASSES OF ENDOGENOUS SMALL RNAS
      • siRNA
      • miRNA
    • OUTLOOK

CHAPTER 4 - RNAI DRUG DELIVERY

  • RNAI DISCOVERED
  • IMPORTANCE OF DELIVERY FOR RNAI THERAPEUTICS
    • DRUG DELIVERY TECHNOLOGIES
      • TABLE 4: ROLE OF DRUG DELIVERY
  • NEED FOR NEW TECHNOLOGIES
  • RNAI DELIVERY-TOOL FOR DRUG DISCOVERY AND DEVELOPMENT
    • siRNA Screening
    • shRNA Screening
  • CHALLENGES TO OVERCOME IN DELIVERING RNAI
    • TABLE 5: DIFFERENT RNAI DELIVERY APPROACHES
  • DIFFERENT DELIVERY TECHNIQUES
    • VECTOR-BASED DELIVERY
    • VECTOR-BASED RNAI FOR IN VIVO RNAI EXPERIMENTS
      • TABLE 6: COMPANIES WITH PLASMID RNAI VECTORS
      • Viral Vectors for Delivery of siRNA
        • TABLE 7: RNAi VIRAL VECTOR DELIVERY METHODS
        • TABLE 8: 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 9: 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 10: SIMPLE EXCIEPIENTS FOR RNAI DELIVERY
    • TRANSFECTION TECHNIQUES
      • Electroporation
      • Conjugates
        • TABLE 11: CONJUGATES, PEPTIDES, AND PROTEINS, OTHERS
      • Peptides and Proteins
      • Dynamic Polyconjugate Transfection
      • Polymer Carriers/Nanoparticles
        • TABLE 12: POLYMER CARRIERS/NANOPARTICLES FOR RNAI DELIVERY
      • Liposomes and Lipoplexes
        • TABLE 13: 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 14: COMPANIES INVOLVED IN DRUG DELIVERY TECHNOLOGIES FOR SIRNA
      • Delivery as Particles or Complexes
      • Delivery with Modifications
    • SUMMARY

CHAPTER 5 - RNAI-BASED THERAPEUTICS

  • RNAI-BASED THERAPEUTICS: THE EMERGING INDUSTRY LANDSCAPE
  • DELIVERING RNAI THERAPEUTICS
    • RNAi and Cancer Therapeutics
  • REGULATORY CONCERNS
  • RNAI IN CLINICAL TRIALS
    • Clinical Trials: RNAi
      • TABLE 15: THE GLOBAL RNA INTERFERENCE THERAPEUTIC PIPELINE
    • Other Candidates in Clinic
    • Terminated Programs
    • Other Programs
  • THERAPEUTIC RNAI DEVELOPMENTS
    • ALN-VSP02
    • Alnylam's ALN-RSV: Respiratory Syncytial Virus
    • ALN-RSV01 Treatment of Respiratory Syncytial Virus Infection in Lung Transplant Patients
      • TABLE 16: RECENT UPDATES: RNAi CLINICAL TRIALS
      • TABLE 17: CLINICAL TRIALS: RNAI-SYSTEMIC DELIVERY
  • CHALLENGES: RNAI-BASED THERAPIES

CHAPTER 6 - 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 18: GLOBAL RNAI RESEARCH SALES AND FORECAST, THROUGH 2018 ($ MILLIONS)
      • FIGURE 1: GLOBAL RNAI RESEARCH SALES, 2010-2018 ($ MILLIONS)
      • TABLE 19: GLOBAL TOOLS, REAGENTS AND THERAPEUTIC MARKET FOR RNAI, THROUGH 2018 ($ MILLIONS)
      • FIGURE 2: GLOBAL TOTAL MARKET FOR RNAI, 2012-2018 ($ 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 20: RNAi COMPANIES WITH TRANSFECTION REAGENTS
      • RNAi Reagents
      • Lipid Transfection
      • In Vivo Reagents and Therapeutics
      • RNAi Experiments in Animals
    • RNAI AND THERAPEUTICS
      • TABLE 21: IN VIVO DELIVERY
      • Viral-based Transfections
        • TABLE 22: VIRAL BASED DELIVERY TECHNOLOGIES
      • Other Methods
      • RNAi Reagents and Kits
        • TABLE 23: RNAI TOOL/REAGENT COMPANIES
    • KEY COMPANIES
      • Inc.Lonza
    • THE FUTURE OF RNAI THERAPEUTICS

CHAPTER 7 - RNAI DRUG DELIVERY-MARKET DATA

  • MARKET TRENDS
  • MARKET SIZE AND GROWTH
    • RNAI DRUGS
    • GLOBAL PHARMACEUTICAL MARKET
      • TABLE 24: GLOBAL PHARMACEUTICAL SALES AND FORECAST THROUGH 2018 ($ BILLIONS)
      • FIGURE 3: GLOBAL PHARMACEUTICAL SALES AND FORECAST, 2011-2018 ($ BILLIONS)
      • Pharmaceutical Growth Rates
      • Emerging Markets
        • TABLE 25: TOTAL UNAUDITED AND AUDITED GLOBAL PHARMACEUTICAL MARKET BY REGION, THROUGH 2017 ($ BILLIONS)
      • U.S. Pharmaceutical Industry
      • Global Pharmaceutical Companies
        • TABLE 26: TOP 25 PHARMACEUTICAL COMPANIES, GLOBAL SALES, 2012 AND 2013 ($ MILLIONS)
        • TABLE 27: TWELVE LARGEST PHARMACEUTICAL COMPANIES 2010 ($ MILLIONS)
  • DRUG DELIVERY TECHNOLOGY LANDSCAPE
    • DRUG DELIVERY MARKET
      • Drug Delivery Technologies and Global Market
        • TABLE 28: TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
        • FIGURE 4: TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
        • TABLE 29: KEY PLAYERS: DRUG DELIVERY TECHNOLOGIES MARKET
      • European Drug Delivery Market
        • TABLE 30: GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS) 123
        • FIGURE 5: GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
      • Oral Drug Delivery
        • TABLE 31: GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
        • FIGURE 6: GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, 2010-2018 ($ MILLIONS)
      • Implantable and Injectable Drug Delivery
        • TABLE 32: GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, THROUGH 2018 ($ MILLIONS)
        • FIGURE 7: GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, 2011-2018 ($ MILLIONS)
      • Transdermal Drug Delivery
        • TABLE 33: GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
        • FIGURE 8: GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
      • Drug Delivery Market in the U.S.
        • TABLE 34: U.S. DRUG DELIVERY TECHNOLOGIES MARKET AND FORECAST THROUGH 2018 ($ MILLIONS)
        • FIGURE 9: U.S. DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • PULMONARY DRUG DELIVERY
      • TABLE 35: TOP ASTHMA AND COPD DRUGS
      • TABLE 36: GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
      • FIGURE 10: GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
  • RNAI MARKET ANALYSIS
    • LEADING PLAYERS
      • Rxi Pharmaceuticals
      • Tekmira Pharmaceuticals
      • Arrowhead Research Corp.
    • RNAI MARKET DYNAMICS
    • RNAI NUCLEIC ACID DRUG DELIVERY
      • TABLE 37: GLOBAL NUCLEIC ACID DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
      • FIGURE 11: GLOBAL NUCLEIC ACID DELIVERY TECHNOLOGIES MARKET, 2010-2018 ($ MILLIONS)
      • TABLE 38: GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2018 ($ MILLIONS)
      • FIGURE 12: GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2018 ($ MILLIONS)
    • NUCLEIC ACID APTAMER-BASED NANOPARTICLES
    • TARGETED DELIVERY
    • THE TOTAL NANOPARTICLE MARKET
      • TABLE 39: GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
      • FIGURE 13: GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, 2012-2018 ($ MILLIONS)
    • DELIVERY OF THERAPEUTICS TO THE TARGET TISSUES
      • TABLE 40: GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
      • FIGURE 14: GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES, MARKET, 2012-2018 ($ MILLIONS)
    • RNAI DELIVERY APPROACHES AND CURRENT DEVELOPMENTS
    • DEVELOPMENT OF RNAI-BASED THERAPEUTICS
    • NANOPARTICLE-BASED DELIVERY
      • RNAi Drugs under Investigation
        • TABLE 41: NON-VIRAL SIRNAS
    • LIPOSOME AND NANOPARTICLES
    • POLYMERS
    • CALANDO PHARMACEUTICALS
    • INTRADIGM
  • MESOPOROUS SILICA NANOPARTICLES
  • PROTEIN OR PEPTIDE-BASED NANOPARTICLES
  • BACTERIOPHAGE BASED NANOPARTICLES
    • SOLSTICE BIOLOGICS
    • GENZYME
      • TABLE 42: KEY RNAI COMPANIES
    • QUARK PHARMACEUTICALS
      • Marina Biotech
      • Lipid and Polymer-Based Systems
        • TABLE 43: LIPID-BASED TECHNOLOGIES-COMPANIES
        • TABLE 44: POLYMER-BASED AND LIPOSOMAL RNAi DELIVERY SYSTEMS (%)
  • RNAI INTO THE PATIENTS
    • ROCHE
      • Polyconjugate Technologies
      • Polymer-Based Systems-Limitations
      • RXi
        • TABLE 45: NANOPARTICLES TECHNOLOGIES-COMPANIES
      • Single siRNA Delivery
        • TABLE 46: SINGLE RNAi DELIVERY TECHNOLOGIES, COMPANIES
  • DICERNA PHARMACEUTICALS
  • CONCLUSIONS

CHAPTER 8 - RNAI DRUG DELIVERY STRATEGIES AND CURRENT MARKET DYNAMICS

  • SYSTEMIC DELIVERY STRATEGIES
  • CHALLENGES WITH RNAI DRUG DELIVERY
  • RNAI THERAPEUTIC MARKET
    • TABLE 47: SELECTED RNAI-BASED DRUGS IN DEVELOPMENT
  • COMMERCIAL POTENTIAL OF RNAI
    • TABLE 48: COMMERCIAL POTENTIAL OF RNAI
  • RNAI'S THERAPEUTIC POTENTIAL
    • HOW DOES RNAI WORK?
    • RNAIS AND PRECLINICAL AND CLINICAL STUDIES
      • CEQ508 in Clinic
      • RNAi as a Therapy
    • GLOBAL MARKET OF THERAPEUTIC RNAI
      • RNAi Therapeutic Market to Play a Key Role by 2020
        • TABLE 49: GLOBAL MARKET FOR THERAPEUTIC RNAi, THROUGH 2018 ($ MILLIONS)
        • FIGURE 15: GLOBAL MARKET FOR THERAPEUTIC RNAi, 2013 AND 2018 ($ MILLIONS)
        • TABLE 50: RNAi THERAPEUTIC COMPANIES
      • Benitec's Clinical Program
        • TABLE 51: ONGOING RNAi CLINICAL TRIALS: NANOPARTICLE DRUG DELIVERY
      • MicroRNA and Market
    • MIRNA MARKET
      • TABLE 52: TOTAL MIRNA MARKET AND FORECAST, THROUGH 2018 ($ MILLIONS)
      • FIGURE 16: TOTAL MIRNA MARKET AND FORECAST, 2013 AND 2018 ($ MILLIONS)
      • TABLE 53: TOTAL MIRNA MARKET SALES, 2008 (%)
  • RECENT DEVELOPMENTS
    • RNAI LICENSING DEALS
      • TABLE 54: MAJOR RNAI LICENSING DEALS, 2005-2010
    • TARGETED DELIVERY
      • TABLE 55: SYSTEMIC DELIVERY TECHNOLOGIES, COMPANIES
    • KEY THERAPEUTIC COMPANIES AND HISTORICAL DEVELOPMENTS
    • ALNYLAM
      • 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 56: ALNYLAM: PIPELINE
    • TEKMIRA
      • Funding
      • Tekmira's LNP Technology
        • TKM-ApoB
      • TKM-PLK1
    • PHASE I/II TKM-PLK1 CLINICAL TRIAL
      • TKM-Ebola
      • Roche
      • Merck
      • Bristol-Myers Squibb
        • TABLE 57: TEKMIRA'S PIPELINE 17
    • HUMAN CLINICAL TRIAL OF TKM-EBOLA
    • RXI PHARMACEUTICALS
      • Pipeline
        • TABLE 58: RXi PHARMACEUTICALS' DEVELOPMENT PIPELINE
      • RXi 109
    • CONNECTIVE TISSUE GROWTH FACTOR (CTGF) AND SCARRING
      • Clinical Program
      • Phase 2
      • RXi Agreements
        • Philips
        • Other Collaborative Agreements
      • Scientific Achievements
      • NIH Grant
    • MARINA BIOTECH
      • Pipeline
        • TABLE 59: MARINA BIOTECH'S DEVELOPMENT PIPELINE
      • Revenue
      • Ongoing Collaborations
        • Beth Israel Deaconess Medical Center-Boston, Mass.
        • Institut Pasteur
        • University of British Columbia (UBC)/Vancouver Prostate Centre (VPC)
        • University of Helsinki
          • TABLE 60: MARINA BIOTECH SELECTED COLLABORATIONS
    • CALANDO PHARMACEUTICALS
      • CALAA-01
    • ISIS PHARMACEUTICALS
      • Pipeline
        • TABLE 61: ISIS PHARMACEUTICALS DRUG DEVELOPMENT PIPELINE
      • Strategic Alliances
      • Pharmaceutical Partners
    • SILENCE THERAPEUTICS
      • Proprietary Delivery Technologies
      • Pipeline
        • TABLE 62: SILENCE THERAPEUTICS, DEVELOPMENT PIPELINE
      • Collaborations
        • Quark Pharmaceuticals
      • Clinical Studies
        • TABLE 63: QUARK PHARMACEUTICALS CURRENT CLINICAL TRIALS
      • Pipeline
        • TABLE 64: QUARK PHARMACEUTICALS-DEVELOPMENT PIPELINE, 2010
      • QPI-1002
    • ROCHE
    • MERCK
    • SIRNAOMICS
      • TABLE 65: SIRNA THERAPEUTIC PROGRAM
    • STP702 (FluQuit): siRNA Therapeutics against Influenza Infection (Pandemic Flu)
    • STP601 (Acurita) siRNA Therapeutics for AMD and Retinopathy
    • STP705 (Cutasil) siRNA Therapeutics for Skin Scarless Wound Healing (SSWH)
    • STP503 (Trisilensa) siRNA Therapeutics for Breast Cancer
  • ACCESS PHARMACEUTICALS
  • KEY MIRNA THERAPEUTIC COMPANIES
    • MIRNA THERAPEUTICS
      • Pipeline
        • TABLE 66: MIRNA, DEVELOPMENT PIPELINE
  • MRX34
    • REGULUS
      • Pipeline
        • TABLE 67: MIRNA PROGRAMS
  • LOCAL THERAPY
  • DRUG DELIVERY IN CANCER
  • RNAI MARKETPLACE CHALLENGES
  • RNAI'S GROWTH AND FUTURE
    • CONCLUSIONS

CHAPTER 9 - COMPANY PROFILES

  • RNAI-DISCOVERY AND GROWTH
  • COMPANY PROFILES
    • ACCESS PHARMACEUTICALS
    • ALNYLAM PHARMACEUTICALS
    • AMBION
    • APARNABIO
    • ARMAGEN TECHNOLOGIES
    • ARROWHEAD RESEARCH
    • ASURAGEN
    • B-BRIDGE INTERNATIONAL
    • BIO-RAD LABORATORIES
    • CALANDO PHARMACEUTICALS
    • CLONTECH LABORATORIES
    • CYNTELLECT
    • DHARMACON
    • DICERNA PHARMACEUTICALS
    • GENECOPOEIA
    • GENETHERA
    • GENLANTIS
    • GENOSENSOR
    • GENERAL RESEARCH LABORATORY
    • IMGENEX
    • INTEGRATED DNA TECHNOLOGIES (IDT)
    • INVITROGEN
    • INVIVOGEN
    • ISIS PHARMACEUTICALS
    • MARINA BIOTECH
    • MERCK
    • MIRAGEN THERAPEUTICS
    • MIRNA THERAPEUTICS INC.
    • NEW ENGLAND BIOLABS
    • OPEN BIOSYSTEMS
    • ORIGENE TECHNOLOGIES
    • PANOMICS
    • PHASERX
    • PROMEGA CORP.
    • PRONAI THERAPEUTICS
    • QUARK PHARMACEUTICALS
    • REGULUS THERAPEUTICS
    • RXI PHARMACEUTICALS
    • SIGMA-ALDRICH
    • SILENCE THERAPEUTICS
    • SIRNAOMICS
    • SOLSTICE BIOLOGICS
    • SOMAGENICS
    • SYSTEM BIOSCIENCES
    • TACERE THERAPEUTICS
    • TEKMIRA PHARMACEUTICALS CORP.
  • COMPANY PROFILES-EUROPE
    • ACTIGENICS
    • AMAXA
    • CENIX BIOSCIENCE
    • EXIQON
    • GENOVIS
    • PROSENSA HOLDING
    • QIAGEN
    • RNAX
    • SANTARIS PHARMA
  • COMPANY PROFILES-ASIA-PACIFIC
    • ALPHAGEN
    • BENITEC
    • BIONEER
      • U.S.
    • CYTOPATHFINDER INC. INC.
    • GENESIS RESEARCH & DEVELOPMENT CORP.
    • KOKEN CO. LTD.
    • ONCOLYS BIOPHARMA INC. INC.
    • SHANGHAI GENEPHARMA CO. LTD.
    • SHANGHAI GENOMICS
    • TRANSGENE BIOTEK

CHAPTER 10 - MARKET SHIFTS IN RNAI SPACE

  • INTELLECTUAL PROPERTY AND PATENT ASPECTS
    • TABLE 68: MAJOR RNAI COMPANIES IN THE U.S.- PATENT DISTRIBUTION
    • FUTURE DEVELOPMENTS
    • SOME SELECTED PATENTS
    • RNAI PLATFORMS
    • SIRNA LITIGATIONS
  • 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
  • CONCLUSIONS

CHAPTER 11 - BIBLIOGRAPHY

LIST OF TABLES

  • SUMMARY TABLE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, THROUGH 2018 ($ MILLIONS)
    • TABLE 1: VARIOUS TECHNOLOGIES FOR GENE SUPPRESSION
    • TABLE 2: THERAPEUTICALLY TARGETED APTAMERS
    • TABLE 3: REQUIRED STEPS FOR DEVELOPING EFFECTIVE RNAI DRUG
    • TABLE 4: ROLE OF DRUG DELIVERY
    • TABLE 5: DIFFERENT RNAI DELIVERY APPROACHES
    • TABLE 6: COMPANIES WITH PLASMID RNAI VECTORS
    • TABLE 7: RNAI VIRAL VECTOR DELIVERY METHODS
    • TABLE 8: LENTIVIRAL DELIVERY PRODUCTS AND COMPANIES
    • TABLE 9: THERAPEUTIC STRATEGIES
    • TABLE 10: SIMPLE EXCIEPIENTS FOR RNAI DELIVERY
    • TABLE 11: CONJUGATES, PEPTIDES, AND PROTEINS, OTHERS
    • TABLE 12: POLYMER CARRIERS/NANOPARTICLES FOR RNAI DELIVERY
    • TABLE 13: LIPOSOMES/LIPOPLEXES AND RNAI DELIVERY
    • TABLE 14: COMPANIES INVOLVED IN DRUG DELIVERY TECHNOLOGIES FOR SIRNA
    • TABLE 15: THE GLOBAL RNA INTERFERENCE THERAPEUTIC PIPELINE
    • TABLE 16: RECENT UPDATES: RNAI CLINICAL TRIALS
    • TABLE 17: CLINICAL TRIALS: RNAI-SYSTEMIC DELIVERY
    • TABLE 18: GLOBAL RNAI RESEARCH SALES AND FORECAST, THROUGH 2018 ($ MILLIONS)
    • TABLE 19: GLOBAL TOOLS, REAGENTS AND THERAPEUTIC MARKET FOR RNAI, THROUGH 2018 ($ MILLIONS)
    • TABLE 20: RNAI COMPANIES WITH TRANSFECTION REAGENTS
    • TABLE 21: IN VIVO DELIVERY
    • TABLE 22: VIRAL BASED DELIVERY TECHNOLOGIES
    • TABLE 23: RNAI TOOL/REAGENT COMPANIES
    • TABLE 24: GLOBAL PHARMACEUTICAL SALES AND FORECAST THROUGH 2018 ($ BILLIONS)
    • TABLE 25: TOTAL UNAUDITED AND AUDITED GLOBAL PHARMACEUTICAL MARKET BY REGION, THROUGH 2017 ($ BILLIONS)
    • TABLE 26: TOP 25 PHARMACEUTICAL COMPANIES, GLOBAL SALES, 2012 AND 2013 ($ MILLIONS)
    • TABLE 27: TWELVE LARGEST PHARMACEUTICAL COMPANIES 2010 ($ MILLIONS)
    • TABLE 28: TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 29: KEY PLAYERS: DRUG DELIVERY TECHNOLOGIES MARKET
    • TABLE 30: GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 31: GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 32: GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 33: GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 34: U.S. DRUG DELIVERY TECHNOLOGIES MARKET AND FORECAST THROUGH 2018 ($ MILLIONS)
    • TABLE 35: TOP ASTHMA AND COPD DRUGS
    • TABLE 36: GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 37: GLOBAL NUCLEIC ACID DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 38: GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 39: GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 40: GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES MARKET, THROUGH 2018 ($ MILLIONS)
    • TABLE 41: NON-VIRAL SIRNAS
    • TABLE 42: KEY RNAI COMPANIES
    • TABLE 43: LIPID-BASED TECHNOLOGIES-COMPANIES
    • TABLE 44: POLYMER-BASED AND LIPOSOMAL RNAI DELIVERY SYSTEMS (%)
    • TABLE 45: NANOPARTICLES TECHNOLOGIES-COMPANIES
    • TABLE 46: SINGLE RNAI DELIVERY TECHNOLOGIES, COMPANIES
    • TABLE 47: SELECTED RNAI-BASED DRUGS IN DEVELOPMENT
    • TABLE 48: COMMERCIAL POTENTIAL OF RNAI
    • TABLE 49: GLOBAL MARKET FOR THERAPEUTIC RNAI, THROUGH 2018 ($ MILLIONS)
    • TABLE 50: RNAI THERAPEUTIC COMPANIES
    • TABLE 51: ONGOING RNAI CLINICAL TRIALS: NANOPARTICLE DRUG DELIVERY
    • TABLE 52: TOTAL MIRNA MARKET AND FORECAST, THROUGH 2018 ($ MILLIONS)
    • TABLE 53: TOTAL MIRNA MARKET SALES, 2008 (%)
    • TABLE 54: MAJOR RNAI LICENSING DEALS, 2005-2010
    • TABLE 55: SYSTEMIC DELIVERY TECHNOLOGIES, COMPANIES
    • TABLE 56: ALNYLAM: PIPELINE
    • TABLE 57: TEKMIRA'S PIPELINE 17
    • TABLE 58: RXI PHARMACEUTICALS' DEVELOPMENT PIPELINE
    • TABLE 59: MARINA BIOTECH'S DEVELOPMENT PIPELINE
    • TABLE 60: MARINA BIOTECH SELECTED COLLABORATIONS
    • TABLE 61: ISIS PHARMACEUTICALS DRUG DEVELOPMENT PIPELINE
    • TABLE 62: SILENCE THERAPEUTICS, DEVELOPMENT PIPELINE
    • TABLE 63: QUARK PHARMACEUTICALS CURRENT CLINICAL TRIALS
    • TABLE 64: QUARK PHARMACEUTICALS-DEVELOPMENT PIPELINE, 2010
    • TABLE 65: SIRNA THERAPEUTIC PROGRAM
    • TABLE 66: MIRNA, DEVELOPMENT PIPELINE
    • TABLE 67: MIRNA PROGRAMS
    • TABLE 68: MAJOR RNAI COMPANIES IN THE U.S.- PATENT DISTRIBUTION
    • TABLE 69: PATENTS

LIST OF FIGURES

  • SUMMARY FIGURE GLOBAL MARKET FOR RNAI DRUG DELIVERY TECHNOLOGIES, 2013-2018 ($ MILLIONS)
    • FIGURE 1: GLOBAL RNAI RESEARCH SALES, 2010-2018 ($ MILLIONS)
    • FIGURE 2: GLOBAL TOTAL MARKET FOR RNAI, 2012-2018 ($ MILLIONS)
    • FIGURE 3: GLOBAL PHARMACEUTICAL SALES AND FORECAST, 2011-2018 ($ BILLIONS)
    • FIGURE 4: TOTAL GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 5: GLOBAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 6: GLOBAL ORAL DRUG DELIVERY TECHNOLOGIES MARKET, 2010-2018 ($ MILLIONS)
    • FIGURE 7: GLOBAL IMPLANTABLE/INJECTABLE DRUG DELIVERY MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 8: GLOBAL TRANSDERMAL DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 9: U.S. DRUG DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 10: GLOBAL PULMONARY DELIVERY TECHNOLOGIES MARKET, 2011-2018 ($ MILLIONS)
    • FIGURE 11: GLOBAL NUCLEIC ACID DELIVERY TECHNOLOGIES MARKET, 2010-2018 ($ MILLIONS)
    • FIGURE 12: GLOBAL APTAMERS THERAPEUTIC MARKET, THROUGH 2018 ($ MILLIONS)
    • FIGURE 13: GLOBAL NANOPARTICLE DRUG DELIVERY TECHNOLOGIES MARKET, 2012-2018 ($ MILLIONS)
    • FIGURE 14: GLOBAL TARGETED DRUG DELIVERY TECHNOLOGIES, MARKET, 2012-2018 ($ MILLIONS)
    • FIGURE 15: GLOBAL MARKET FOR THERAPEUTIC RNAI, 2013 AND 2018 ($ MILLIONS)
    • FIGURE 16: TOTAL MIRNA MARKET AND FORECAST, 2013 AND 2018 ($ MILLIONS)
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