TABLE OF CONTENTS
1 INTRODUCTION 21
1.1 OBJECTIVES OF THE STUDY 21
1.2 MARKET DEFINITION 21
1.3 OVERVIEW OF THE GLOBAL GENE THERAPY MARKET 21
1.4 CURRENCY AND PRICING 23
1.5 LIMITATIONS 23
1.6 MARKETS COVERED 23
2 MARKET SEGMENTATION 25
2.1 MARKETS COVERED 25
2.2 GEOGRAPHICAL SCOPE 26
2.3 YEARS CONSIDERED FOR THE STUDY 27
2.4 DBMR TRIPOD DATA VALIDATION MODEL 28
2.5 PRIMARY INTERVIEWS WITH KEY OPINION LEADERS 31
2.6 MULTIVARIATE MODELLING 32
2.7 MARKET END USER COVERAGE GRID 32
2.8 PRODUCT LIFELINE CURVE 33
2.9 DBMR MARKET POSITION GRID 34
2.10 VENDOR SHARE ANALYSIS 35
2.11 SECONDARY SOURCES 36
2.12 ASSUMPTIONS 36
3 EXECUTIVE SUMMARY 37
4 PREMIUM INSIGHTS 40
4.1 PESTEL ANALYSIS 43
4.2 PORTER FIVE ANALYSIS 44
5 UPDATE ON GERMLINE GENE THERAPY 45
5.1 GERMLINE GENE THERAPY 45
6 GLOBAL GENE THERAPY MARKET, MO 46
6.1 DRIVERS 48
6.1.1 NOVEL APPROACHES TO GENE THERAPY 48
6.1.2 INCREASING PREVALENCE OF GENETIC DISORDERS 48
6.1.3 THE GROWING INVESTMENT BY BIOTECHNOLOGY & PHARMACEUTICAL COMPANIES 49
6.1.4 GROWING DEMAND FOR PERSONALIZED MEDICINE 50
6.2 RESTRAINTS 50
6.2.1 HIGH COST OF GENE THERAPY 50
6.2.2 ETHICAL AND SAFETY CONCERNS 51
6.2.3 COMPLEXITY OF GENE THERAPY 51
6.3 OPPORTUNITIES 52
6.3.1 RISE IN STRATEGIC ACQUISITION AND PARTNERSHIP AMONG ORGANIZATIONS 52
6.3.2 RISING APPROVAL FOR GENE THERAPY PRODUCTS 52
6.4 CHALLENGES 53
6.4.1 STRINGENT REGULATIONS FOR GENE THERAPY PRODUCTS 53
6.4.2 LONG-TERM SAFETY AND EFFICACY 53
7 GLOBAL GENE THERAPY MARKET, BY VECTOR TYPE 54
7.1 OVERVIEW 55
7.2 VIRAL VECTOR 58
7.2.1 ADENOVIRUS 59
7.2.2 RETROVIRUS 59
7.2.3 LENTIVIRUS 59
7.2.4 ADENO-ASSOCIATED VIRUS 59
7.2.5 VACCINIA VIRUS 59
7.2.6 HERPES SIMPLEX VIRUS 59
7.2.7 OTHERS 59
7.3 NON-VIRAL VECTOR 59
7.3.1 LIPOFECTION 60
7.3.2 INJECTION OF NAKED DNA 60
8 GLOBAL GENE THERAPY MARKET, BY METHOD 61
8.1 OVERVIEW 62
8.2 EX-VIVO 65
8.3 IN-VIVO 65
9 GLOBAL GENE THERAPY MARKET, BY APPLICATION 67
9.1 OVERVIEW 68
9.2 ONCOLOGICAL DISORDERS 71
9.3 CARDIOVASCULAR DISEASES 71
9.4 INFECTIOUS DISEASES 72
9.5 RARE DISEASES 73
9.6 NUEROLOGICAL DISORDERS 74
9.7 OTHER DISEASES 75
10 GLOBAL GENE THERAPY MARKET, BY END USER 76
10.1 OVERVIEW 77
10.2 CANCER INSTITUTES 80
10.3 HOSPITALS 80
10.4 RESEARCH INSTITUTES 81
10.5 OTHERS 82
11 GLOBAL GENE THERAPY MARKET, BY REGION 83
11.1 OVERVIEW 84
11.2 NORTH AMERICA 89
11.2.1 U.S. 95
11.2.2 CANADA 97
11.2.3 MEXICO 99
11.3 EUROPE 101
11.3.1 GERMANY 107
11.3.2 FRANCE 109
11.3.3 U.K. 111
11.3.4 ITALY 113
11.3.5 SPAIN 115
11.3.6 RUSSIA 117
11.3.7 TURKEY 119
11.3.8 NETHERLANDS 121
11.3.9 BELGIUM 123
11.3.10 SWITZERLAND 125
11.3.11 REST OF EUROPE 127
11.4 ASIA-PACIFIC 128
11.4.1 CHINA 134
11.4.2 JAPAN 136
11.4.3 SOUTH KOREA 138
11.4.4 INDIA 140
11.4.5 AUSTRALIA 142
11.4.6 SINGAPORE 144
11.4.7 THAILAND 146
11.4.8 MALAYSIA 148
11.4.9 INDONESIA 150
11.4.10 PHILIPPINES 152
11.4.11 REST OF ASIA-PACIFIC 154
11.5 SOUTH AMERICA 155
11.5.1 BRAZIL 161
11.5.2 REST OF SOUTH AMERICA 163
11.6 MIDDLE EAST AND AFRICA 164
11.6.1 SOUTH AFRICA 170
11.6.2 REST OF MIDDLE EAST AND AFRICA 172
12 GLOBAL GENE THERAPY MARKET, COMPANY LANDSCAPE 173
12.1 COMPANY SHARE ANALYSIS: GLOBAL 173
12.2 COMPANY SHARE ANALYSIS: NORTH AMERICA 174
12.3 COMPANY SHARE ANALYSIS: EUROPE 175
12.4 COMPANY SHARE ANALYSIS: ASIA-PACIFIC 176
13 COMPANY PROFILES 177
13.1 BIOGEN 177
13.1.1 COMPANY SNAPSHOT 177
13.1.2 REVENUE ANALYSIS 177
13.1.3 COMPANY SHARE ANALYSIS 178
13.1.4 SWOT ANALYSIS 178
13.1.5 PRODUCT PORTFOLIO 179
13.1.6 RECENT DEVELOPMENT 179
13.2 KITE PHARMA 180
13.2.1 COMPANY SNAPSHOT 180
13.2.2 REVENUE ANALYSIS 180
13.2.3 COMPANY SHARE ANALYSIS 181
13.2.4 SWOT ANALYSIS 181
13.2.5 PRODUCT PORTFOLIO 182
13.2.6 RECENT DEVELOPMENT 182
13.3 NOVARTIS AG 183
13.3.1 COMPANY SNAPSHOT 183
13.3.2 REVENUE ANALYSIS 183
13.3.3 COMPANY SHARE ANALYSIS 184
13.3.4 SWOT ANALYSIS 184
13.3.5 PRODUCT PORTFOLIO 185
13.3.6 RECENT DEVELOPMENTS 185
13.4 BRISTOL-MYERS SQUIBB COMPANY. 186
13.4.1 COMPANY SNAPSHOT 186
13.4.2 REVENUE ANALYSIS 186
13.4.3 COMPANY SHARE ANALYSIS 187
13.4.4 SWOT ANALYSIS 187
13.4.5 PRODUCT PORTFOLIO 188
13.4.6 RECENT DEVELOPMENT 188
13.5 OXFORD BIOMEDICA 189
13.5.1 COMPANY SNAPSHOT 189
13.5.2 REVENUE ANALYSIS 189
13.5.3 COMPANY SHARE ANALYSIS 190
13.5.4 SWOT ANALYSIS 190
13.5.5 PRODUCT PORTFOLIO 191
13.5.6 RECENT DEVELOPMENTS 191
13.6 AGC BIOLOGICS 192
13.6.1 COMPANY SNAPSHOT 192
13.6.2 PRODUCT PORTFOLIO 192
13.6.3 RECENT DEVELOPMENT 192
13.7 ANGES, INC 193
13.7.1 COMPANY SNAPSHOT 193
13.7.2 REVENUE ANALYSIS 193
13.7.3 PRODUCT PORTFOLIO 194
13.7.4 RECENT DEVELOPMENT 194
13.8 AMGEN INC. 195
13.8.1 COMPANY SNAPSHOT 195
13.8.2 REVENUE ANALYSIS 195
13.8.3 PRODUCT PORTFOLIO 196
13.8.4 RECENT DEVELOPMENT 196
13.9 BLUEBIRD BIO, INC. 197
13.9.1 COMPANY SNAPSHOT 197
13.9.2 PRODUCT PORTFOLIO 197
13.9.3 RECENT DEVELOPMENT 197
13.10 CHIESI FARMACEUTICI S.P.A 198
13.10.1 COMPANY SNAPSHOT 198
13.10.2 REVENUE ANALYSIS 198
13.10.3 PRODUCT PORTFOLIO 199
13.10.4 RECENT DEVELOPMENT 199
13.11 DENDREON PHARMACEUTICALS LLC 200
13.11.1 COMPANY SNAPSHOT 200
13.11.2 PRODUCT PORTFOLIO 200
13.11.3 RECENT DEVELOPMENT 200
13.12 ENZYVANT THERAPEUTICS GMBH 201
13.12.1 COMPANY SNAPSHOT 201
13.12.2 RODUCT PORTFOLIO 201
13.12.3 RECENT DEVELOPMENT 201
13.13 FERRING B.V. 202
13.13.1 COMPANY SNAPSHOT 202
13.13.2 PRODUCT PORTFOLIO 202
13.13.3 RECENT DEVELOPMENT 202
13.14 JANSSEN PHARMACEUTICALS, INC. 203
13.14.1 COMPANY SNAPSHOT 203
13.14.2 PRODUCT PORTFOLIO 203
13.14.3 RECENT DEVELOPMENT 203
13.15 MALLINCKRODT. 204
13.15.1 COMPANY SNAPSHOT 204
13.15.2 REVENUE ANALYSIS 204
13.15.3 PRODUCT PORTFOLIO 205
13.15.4 RECENT DEVELOPMENT 205
13.16 ORCHARD THERAPEUTICS PLC. 206
13.16.1 COMPANY SNAPSHOT 206
13.16.2 REVENUE ANALYSIS 206
13.16.3 PRODUCT PORTFOLIO 207
13.16.4 RECENT DEVELOPMENT 207
13.17 SHANGHAI SUNWAY BIOTECH CO., LTD. 208
13.17.1 COMPANY SNAPSHOT 208
13.17.2 PRODUCT PORTFOLIO 208
13.17.3 RECENT DEVELOPMENT 208
13.18 SIBONO 209
13.18.1 COMPANY SNAPSHOT 209
13.18.2 PRODUCT PORTFOLIO 209
13.18.3 RECENT DEVELOPMENT 209
13.19 SPARK THERAPEUTICS, INC. 210
13.19.1 COMPANY SNAPSHOT 210
13.19.2 PRODUCT PORTFOLIO 210
13.19.3 RECENT DEVELOPMENT 210
13.20 UNIQURE NV. 211
13.20.1 COMPANY SNAPSHOT 211
13.20.2 REVENUE ANALYSIS 211
13.20.3 PRODUCT PORTFOLIO 212
13.20.4 RECENT DEVELOPMENT 212
14 QUESTIONNAIRE 213
15 RELATED REPORTS 216

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