遺伝子治療の世界市場分析...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 6
1.1 List of Tables 9
1.2 List of Figures 10

2 Introduction 12
2.1 Report Scope 12
2.2 Companies Mentioned 13
2.3 Upcoming Reports 13
2.4 Recently Published Reports 13

3 Gene Therapy Overview 14
3.1 What is Gene Therapy? 14
3.2 Gene Delivery 16
3.2.1 Viral Vectors 17
3.2.2 Non-Viral Vectors and Naked DNA 21

4 Market Developments 22
4.1 Turning Point: Glybera Approval 23
4.2 GSK Files Application to the EMA for Gene Therapy for ADA-SCID 24
4.3 Celladon’s Reports Negative Results for Mydicar in Advanced Heart Failure 25
4.4 Spark Initiates Phase I/II Clinical Trial of SPK-CHM for Choroideremia 26
4.5 FDA Accepts Sangamo’s IND to Begin Trial of Gene Therapy for Beta-Thalassemia 26
4.6 Paying for Gene Therapies: Annuity Model 27

5 Clinical Trials 29
5.1 Overview 29
5.2 Company 31
5.3 Phase of Development 32
5.4 Therapy Area 33
5.5 Indication 35
5.6 Investigators 36
5.6.1 Specialty 36
5.6.2 Country 38

6 Company Analysis 39
6.1 Overview 39
6.1.1 Entity Type 39
6.1.2 Regional Split 39
6.2 Financial Highlights 40
6.2.1 Revenue 41
6.2.2 R&D Spending 42
6.2.3 Market Capitalization 43
6.2.4 Stock Performance 45
6.3 Pipeline Profiles 46
6.3.1 Advantagene 47
6.3.2 ALS Therapy Development Institute 48
6.3.3 Amarna Therapeutics 49
6.3.4 American Gene Technologies International 50
6.3.5 AnGes MG 51
6.3.6 Applied Genetic Technologies Corporation 54
6.3.7 Asklepios BioPharmaceutical 55
6.3.8 Avalanche Biotechnologies 56
6.3.9 Bluebird bio 58
6.3.10 Celladon 60
6.3.11 Dimension Therapeutics 61
6.3.12 GeneCure Biotechnologies 62
6.3.13 Genethon 63
6.3.14 GenSight Biologics 63
6.3.15 Oxford BioMedica 64
6.3.16 ReGenX Biosciences 66
6.3.17 Sangamo BioSciences 67
6.3.18 Spark Therapeutics 70
6.3.19 UniQure 71
6.3.20 Voyager Therapeutics 74

7 Deals Landscape 75
7.1 Overview 75
7.1.1 Geography 77
7.1.2 Entity Type 79
7.1.3 Company 80
7.1.4 Stage 81
7.1.5 Therapy Area 82
7.1.6 Indication 84
7.1.7 Mechanism of Action 86
7.2 Deal Type 86
7.2.1 Mergers and Acquisitions 87
7.2.2 Licensing and Partnerships 89
7.2.3 Capital Raisings 92

8 Strategic Outlook 95

9 Appendix 96
9.1 Bibliography 96
9.2 Research Methodology 96
9.2.1 Coverage 96
9.2.2 Secondary Research 97
9.3 About the Author 98
9.3.1 Adam Dion, Senior Industry Analyst 98
9.4 Global Head of Healthcare 99
9.5 About the Industry Dynamics Team 99
9.6 About GlobalData 100
9.7 Disclosure Information 100
9.8 Disclaimer 100