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遺伝子改変治療の臨床用途:遺伝子治療、遺伝子改変細胞治療、RNA治療、遺伝子編集

Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

発行 BCC Research 商品コード 694192
出版日 ページ情報 英文 211 Pages
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遺伝子改変治療の臨床用途:遺伝子治療、遺伝子改変細胞治療、RNA治療、遺伝子編集 Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing
出版日: 2018年08月31日 ページ情報: 英文 211 Pages
概要

世界における遺伝子改変治療市場は、2018年の23億米ドルから、2023年までに174億米ドルへ達すると予測され、予測期間中は49.9%のCAGR (年間複合成長率) が見込まれています。

当レポートでは、世界の遺伝子改変治療市場を調査し、市場概要、技術・用途区分別の動向と市場規模の推移と予測、特許動向、主要企業のプロファイルなどをまとめています。

第1章 イントロダクション

第2章 サマリー・ハイライト

第3章 概要

  • 調査範囲
  • 調査対象の遺伝子改変治療
  • 遺伝子改変治療プラットフォーム技術
  • 遺伝子改変治療の成長促進要因
  • 世界の遺伝子改変治療市場
  • 製品・技術のライフサイクル状況
  • 遺伝子改変治療産業

第4章 技術

  • 遺伝子技術のイントロダクション
  • 遺伝的変異・分析
  • 遺伝子改変治療
  • 遺伝子治療技術
  • RNA治療技術
  • 遺伝子編集技術
  • 遺伝子改変細胞治療技術
  • デリバリー技術
  • イントロダクション
  • ウィルスデリバリー技術
  • 非ウィルスデリバリー技術
  • 遺伝子治療薬の設計・製造

第5章 遺伝子改変治療用途

第6章 遺伝子改変治療産業

  • サマリー
  • 遺伝子編集産業
  • 遺伝子治療産業
  • 遺伝子改変細胞療法産業
  • RNA治療産業
  • レンチウイルス製造業

第7章 買収・戦略的アライアンス

第8章 遺伝子改変治療市場

  • 成長促進要因
  • ペイメント・ビジネスモデル
  • 遺伝子改変治療市場
  • 遺伝子改変治療:プラットフォーム技術別
  • 遺伝子改変治療:疾病分類別
  • 遺伝子改変治療:デリバリー技術別
  • 遺伝子改変治療:適応症別
  • 遺伝子改変治療:地域別

第9章 特許

  • 遺伝子編集関連特許
  • 遺伝子編集特許:Sangamo社のケーススタディ
  • 遺伝子治療デリバリー特許
  • RNA治療関連特許
  • CAR T-細胞特許

第10章 企業プロファイル

図表

List of Tables

  • Summary Table: Global Market for Genetic Modification Therapies, by Disease Category, Through 2023
  • Table 1: Scope of Report
  • Table 2: Genetic Modification Therapies Covered in Report
  • Table 3: Genetic Modification Platforms
  • Table 4: Key Market Growth Driving Forces of Genetic Modification Therapies
  • Table 5: Global Market for Genetic Modification Therapies, by Delivery Technology, Through 2023
  • Table 6: Genetic Modification Therapy Technology Life-Cycle Stage
  • Table 7: Characteristics of the Genetic Modification Therapies Industry
  • Table 8: Chemical Building Blocks of DNA and RNA
  • Table 9: DNA Translation and Transcription
  • Table 10: Long-range DNA Structure
  • Table 11: Genetic Variant Types
  • Table 12: Therapeutic Intervention Platforms
  • Table 13: Genetic Modification Approaches
  • Table 14: Distinct Features of the Genetic Modification Approaches
  • Table 15: Main Components of Gene Therapy Technology
  • Table 16: Gene Therapy Delivery Technology Parameters
  • Table 17: Clinical Criteria for Gene Therapy
  • Table 18: RNA Therapy Segments
  • Table 19: Gene Editing Therapeutic Interventions
  • Table 20: Gene Editing Technology Approaches
  • Table 21: Comparison of the Major Gene Editing Technologies
  • Table 22: Genetically Modified Cell Therapy Technologies
  • Table 23: Delivery Vector Technical Hurdles
  • Table 24: Comparison of Viral Vector Types
  • Table 25: Nonviral Delivery Methods
  • Table 26: Steps in Designing and Manufacturing a Gene Therapy Medicine
  • Table 27: Near-Term Applications for Genetic Modification Therapies
  • Table 28: Characteristics of Liver Organ
  • Table 29: Sectors within the Genetic Modification Therapies Industry
  • Table 30: Gene Editing Industry
  • Table 31: Gene Therapy Companies
  • Table 32: Genetically Modified Cell Therapy Clinical Industry
  • Table 33: RNA Therapy Companies
  • Table 34: GMP Clinical-Grade Lentiviral Manufacturing Companies
  • Table 35: Genetic Modification Therapies Industry Acquisitions, Jan. 2016-June 2018
  • Table 36: Genetic Modification Therapies Industry Strategic Alliances, Jan. 2016-June 2018
  • Table 37: Genetic Modification Therapies Key Market Growth Driving Forces
  • Table 38: Global Market for Genetic Modification Therapies, by Platform Technology, Through 2023
  • Table 39: Global Market for Gene Editing Therapies, by Disease Category, Through 2023
  • Table 40: Global Market for Gene Editing Therapies, by Technology, Through 2023
  • Table 41: Global Market for Gene Editing Therapies, by Delivery Type, Through 2023
  • Table 42: CRISPR Clinical Pipeline
  • Table 43: Global Market for Gene Therapy, by Disease Category, Through 2023
  • Table 44: Inherited Retinal Diseases
  • Table 45: Global Market for Gene Therapy, by Platform Technology, Through 2023
  • Table 46: Global Market for Gene Therapy, by Delivery Technology, Through 2023
  • Table 47: Gene Therapy AAV Vector Clinical Landscape
  • Table 48: Global Market for Genetically Modified Cell Therapy, by Disease Category, Through 2023
  • Table 49: Global Market for Genetically Modified Cell Therapy, by Platform Technology, Through 2023
  • Table 50: Global Genetically Modified Cell Therapy Market, by Delivery Technology, Through 2023
  • Table 51: Allogeneic versus Autologous Approaches
  • Table 52: Allogeneic Clinical and Preclinical Cell Therapy Programs
  • Table 53: Global Market for RNA Therapy, by Disease Category, Through 2023
  • Table 54: Global Market for RNA Therapy, by Platform Technology, Through 2023
  • Table 55: GalNAc-conjugated RNA Therapy Pipeline
  • Table 56: Global Market for RNA Therapy, by Delivery Technology, Through 2023
  • Table 57: Global Market for Genetic Modification Therapies, by Disease Category, Through 2023
  • Table 58: Indications Driving Market Growth
  • Table 59: Global Market for Genetic Modification Therapies Market, by Delivery Technology, Through 2023
  • Table 60: Global Market for Genetic Modification Medicines: Marketed and Late-Stage Pipeline
  • Table 61: Global Market for Genetic Modification Therapies in Cardiology, by Indication, Through 2023
  • Table 62: Global Market for Genetic Modification Therapies in Oncology, by Indication, Through 2023
  • Table 63: Barriers to CAR T-Cell Solid Tumor Development
  • Table 64: CAR T-Cell Solid Tumor Development Status
  • Table 65: Global Market for Genetic Modification Therapies in Ophthalmology, by Indication, Through 2023
  • Table 66: Global Market for Genetic Modification Therapies in Hematology, by Indication, Through 2023
  • Table 67: Hemophilia A and B: Drugs in Development
  • Table 68: Global Market for Genetic Modification Therapies in Musculoskeletal Applications, by Indication, Through 2023
  • Table 69: Global Market for Genetic Modification Therapies, by Indication, Through 2023
  • Table 70: Global Market for Genetic Modification Therapies in Rare Diseases, by Indication, Through 2023
  • Table 71: Global Market for Genetic Modification Therapies in Other Applications, by Indication, Through 2023
  • Table 72: Global Market for Genetic Modification Therapies, by Region, Through 2023
  • Table 73: Global Market for Gene Editing Therapy, by Region, Through 2023
  • Table 74: Global Market for Gene Therapy, by Region, Through 2023
  • Table 75: Global Market for Genetically Modified Cell Therapy, by Region, Through 2023
  • Table 76: Global Market for RNA Therapy, by Region, Through 2023
  • Table 77: CRISPR-Cas9 Patent Groups
  • Table 78: CRISPR-Cas9 Patent Licensing Fields
  • Table 79: Sangamo's Collaboration Agreements Relating to ZFN Technology
  • Table 80: Sangamo Therapeutics' Patent Position
  • Table 81: Oxford BioMedica's Key Patents
  • Table 82: RNA Therapy Patents and Patent Applications, by Leading Companies
  • Table 83: CAR T-cell Patentable Technologies
  • Table 84: Affimers versus Antibodies for Gene Therapy Applications
  • Table 85: Gene Editing versus Gene Therapy Approach

List of Figures

  • Summary Figure: Global Market for Genetic Modification Therapies, by Disease Category, 2017-2023
  • Figure 1: Complexity of Therapeutic Approaches
  • Figure 2: Main Principle of Molecular Biology
目次
Product Code: BIO159A

Report Highlights:

The global market for genetic modification therapies should grow from $2.3 billion in 2018 to reach $17.4 billion by 2023, growing at a compound annual growth rate (CAGR) of 49.9% during the forecast period of 2018-2023.

Neurology market for genetic modification therapies should grow from $1.5 billion in 2018 to reach $3.2 billion by 2023, growing at a CAGR of 16.9% during the forecast period of 2018-2023.

Rare diseases market for genetic modification therapies should grow from $51.8 million in 2018 to reach $2.6 billion by 2023, growing at a CAGR of 119.6% during the forecast period of 2018-2023.

Report Scope:

The study scope includes clinical therapies using gene editing, gene therapy, genetically modified cell therapy (chimeric antigen receptor T-cells, or CAR T-cells) or RNA therapy platforms. Delivery technologies covered include AAV, adenovirus, lentivirus, retrovirus, other viral and nonviral. Disease areas covered include cardiology, oncology, ophthalmology, hematology, musculoskeletal, neurology, rare diseases and others. Therapies that are already commercialized or are forecast to be commercialized with the next five years are included.

We analyze key genetic modification therapy technologies and products to determine present and future market status and forecasted growth from 2018 through 2023. We also discuss strategic alliances, industry structures, competitive dynamics, patents and market driving forces.

BCC Research examines the genetic modification therapies industry by market segment, including the following segments: gene editing, gene therapy, CAR T-cell therapy, RNA therapy and lentiviral production. The geographic market for genetic modification therapy products by platform technology is analyzed. Regions include North America, Europe, Asia-Pacific and the rest of the world.

The role of key strategic alliances and acquisitions from Jan. 2016 through June 2018 is discussed.

The challenges involved with pricing for genetic modification therapies that can offer one-time cures for diseases is discussed. Emerging markets for clinical gene editing are analyzed. More than 145 companies in the genetic modification therapies industry are profiled.

Report Includes:

  • 30 data tables and 56 additional tables
  • An overview of the global market for genetic modification therapies
  • Analyses of global market trends with data from 2017, estimates for 2018, and projections of compound annual growth rates (CAGRs) through 2023
  • Information on the genetic variation analysis and genetic modification technologies
  • Evaluation of key growth driving forces, acquisitions and strategic alliances related to the market
  • Detailed description of the criteria for selecting gene modification therapy applications
  • Review of patents for gene editing, gene therapy, RNA therapy and gene modification therapies
  • Profiles of the key companies in the market, including 4d Molecular Therapeutics LLC, Abeona Therapeutics LLC, Acer Therapeutics Inc., Allergan Plc and American Gene Technologies International Inc.

Table of Contents

Chapter 1 Introduction

  • Study Goals and Objectives
  • Reasons for Doing the Study
  • Contribution of the Study and for Whom
  • Scope and Format
  • Methodology
  • Information Sources
  • Geographical Breakdown
  • Analyst's Credentials
  • BCC Custom Research
  • Related BCC Research Reports

Chapter 2 Summary and Highlights

Chapter 3 Overview

  • Scope of Report
  • Genetic Modification Therapies Covered in Report
  • Genetic Modification Therapy Platform Technologies
  • Forces Driving the Growth of the Market for Genetic Modification Therapies
  • Global Market for Genetic Modification Therapies
  • Life-Cycle Status of Products and Technologies
  • Genetic Modification Therapies Industry

Chapter 4 Technology

  • Introduction to Genetic Technology
  • Genetic Variation and Analysis
  • Genetic Modification Therapies
  • Gene Therapy Technologies
  • RNA Therapy Technologies
  • Gene Editing Technologies
  • Genetically Modified Cell Therapy Technologies
  • Delivery Technologies
  • Introduction
  • Viral Delivery Technologies
  • Nonviral Delivery Technologies
  • Design and Manufacture of a Gene Therapy Medicine

Chapter 5 Genetic Modification Therapy Applications

Chapter 6 Genetic Modification Therapies Industry

  • Summary
  • Gene Editing Industry
  • Gene Therapy Industry
  • Genetically Modified Cell Therapy Industry
  • RNA Therapy Industry
  • Lentivirus Production Industry

Chapter 7 Acquisitions and Strategic Alliances

  • Acquisitions
  • Strategic Alliances

Chapter 8 Markets for Genetic Modification Therapies

  • Growth Driving Forces
  • Payment and Business Models for Genetic Modification Therapeutics
  • Market for Genetic Modification Therapies
  • Genetic Modification Therapies by Platform Technology
  • Genetic Modification Therapies by Disease Category
  • Genetic Modification Therapies by Delivery Technology
  • Genetic Modification Therapies by Indication
  • Genetic Modification Therapies by Region

Chapter 9 Patents

  • Gene Editing-Related Patents
  • Gene Editing Patents-Sangamo Case Study
  • Gene Therapy Delivery Patents
  • RNA Therapy-Related Patents
  • CAR T-cell Patents

Chapter 10 Company Profiles

  • 4D MOLECULAR THERAPEUTICS
  • ABCLON
  • ABEONA THERAPEUTICS LLC
  • ACER THERAPEUTICS INC.
  • ADAPTIMMUNE THERAPEUTICS
  • ADVANTAGENE INC.
  • ADVERUM BIOTECHNOLOGIES
  • AEVI GENOMIC MEDICINE INC.
  • AGILIS BIOTHERAPEUTICS
  • ALDEVRON
  • ALLERGAN PLC
  • ALLOGENE THERAPEUTICS INC.
  • AMERICAN GENE TECHNOLOGIES INTERNATIONAL INC.
  • AMGEN INC.
  • ANGES INC.
  • APEIRON BIOLOGICS AG
  • APPLIED GENETIC TECHNOLOGIES CORP.
  • ARBOR BIOTECHNOLOGIES INC.
  • ARCTURUS THERAPEUTICS INC.
  • ASKLEPIOS BIOPHARMACEUTICAL INC.
  • ASTELLAS PHARMA INC.
  • AUDENTES THERAPEUTICS INC.
  • AVACTA GROUP PLC
  • AVROBIO INC.
  • BEAM THERAPEUTICS INC.
  • BELLICUM PHARMACEUTICALS INC.
  • BINEX CO. LTD.
  • BIOCUREPHARM CORP.
  • BIOGEN INC.
  • BIOMARIN PHARMACEUTICAL INC.
  • BLUEBIRD BIO
  • B-MOGEN BIOTECHNOLOGIES INC.
  • BRAMMER BIO
  • CARIBOU BIOSCIENCES
  • CASEBIA THERAPEUTICS
  • CATHERICS PTY LTD.
  • CELGENE CORP.
  • CELLECTIS SA
  • CELL MEDICA LTD.
  • CELLULAR BIOMEDICINE GROUP
  • CELYAD
  • COGNATE BIOSERVICES
  • COMBIGENE AB
  • CRISPR THERAPEUTICS AG
  • CSL BEHRING LLC
  • CUROCELL CO. LTD.
  • EDITAS MEDICINE INC.
  • ENCODED GENOMICS INC.
  • ERRANT GENE THERAPEUTICS LLC
  • ESTEVE
  • ETAGEN PHARMA
  • EUTILEX CO. LTD.
  • FATE THERAPEUTICS INC.
  • FERRING PHARMACEUTICALS SA
  • FIBROCELL SCIENCE
  • FKD THERAPIES OY
  • FORTRESS BIOTECH INC.
  • FREELINE THERAPEUTICS
  • GE HEALTHCARE
  • GENE BIOTHERAPEUTICS INC.
  • GENENTA SCIENCE SRL
  • GENERATION BIO
  • GENETHON SA
  • GENSIGHT BIOLOGICS SA
  • GILEAD SCIENCES INC.
  • GLAXOSMITHKLINE PLC
  • GLOBAL BIOTHERAPEUTICS USA INC.
  • GREEN CROSS CELL CORP.
  • HEMERA BIOSCIENCES INC.
  • HITACHI CHEMICAL ADVANCED THERAPEUTICS LLC
  • HOMOLOGY MEDICINES INC.
  • HORAMA SA
  • IMMUNOCHINA PHARMACEUTICALS
  • IMMUNOCORE LTD.
  • INDEE LABS INC.
  • INTELLIA THERAPEUTICS INC.
  • INTREXON CORP.
  • JIVANA BIOTECHNOLOGY
  • JOHNSON & JOHNSON
  • JUVENTAS THERAPEUTICS INC.
  • KOLON LIFE SCIENCES INC.
  • KRYSTAL BIOTECH
  • KUBOTA PHARMACEUTICAL HOLDINGS CO. LTD.
  • LIMELIGHT BIO INC.
  • LOGICBIO THERAPEUTICS INC.
  • LONZA GROUP LTD.
  • LYSOGENE SA
  • MARCALA BIOTECH INC.
  • MAXCYTE INC.
  • MEDIGENE AG
  • MEIRA GTX
  • MERCK & CO.
  • MERCK KGAA
  • MESOBLAST LTD.
  • MILLIPORE SIGMA
  • MILO BIOTECHNOLOGY LLC
  • MOLMED SPA
  • MORPHOGENESIS INC.
  • MYONEXUS THERAPEUTICS INC.
  • NANTKWEST INC.
  • NEXIMMUNE INC.
  • NEWLINK GENETICS CORP.
  • NIGHTSTAR THERAPEUTICS PLC
  • NOVARTIS PHARMA AG
  • NOVASEP LLC
  • OCUNEXUS THERAPEUTICS INC.
  • ORCHARD THERAPEUTICS
  • OXFORD BIOMEDICA PLC
  • PEPROMENE BIO INC.
  • PERIPHAGEN INC.
  • PFIZER INC.
  • POSEIDA THERAPEUTICS INC.
  • PRECISION BIOSCIENCE
  • REGENXBIO INC.
  • RENOVA THERAPEUTICS
  • ROCKET PHARMA LTD.
  • RXI PHARMACEUTICALS CORP.
  • SANGAMO THERAPEUTICS INC.
  • SANOFI SA
  • SAREPTA THERAPEUTICS INC.
  • SELECTA BIOSCIENCES INC.
  • SENTI BIOSCIENCES
  • SHIRE PLC
  • SIBIONO GENETECH CO. LTD.
  • SILLAGEN BIOTHERAPEUTICS
  • SOLID BIOSCIENCES LLC
  • SPARK THERAPEUTICS INC.
  • SYNPROMICS LTD.
  • TAKARA BIO
  • THERAGENE PHARMACEUTICALS INC.
  • THERMO FISHER SCIENTIFIC
  • TOCAGEN
  • TOOLGEN
  • TRANSLATE BIO
  • TRANSGENE SA
  • TX CELL
  • ULTRAGENYX PHARMACEUTICAL INC.
  • UNIQURE NV
  • UNUM THERAPEUTICS INC.
  • VASCULAR BIOGENICS LTD.
  • VIRACTA THERAPEUTICS
  • VIROMED CO. LTD.
  • VIVET THERAPEUTICS
  • VOYAGER THERAPEUTICS INC.
  • WUXI APPTECH
  • ZIOPHARM ONCOLOGY
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