TABLE OF CONTENTS
1 INTRODUCTION 20
1.1 OBJECTIVES OF THE STUDY 20
1.2 MARKET DEFINITION 20
1.3 OVERVIEW OF THE NORTH AMERICA GENE THERAPY MARKET 20
1.4 CURRENCY AND PRICING 22
1.5 LIMITATIONS 22
1.6 MARKETS COVERED 22
2 MARKET SEGMENTATION 24
2.1 MARKETS COVERED 24
2.2 GEOGRAPHICAL SCOPE 25
2.3 YEARS CONSIDERED FOR THE STUDY 26
2.4 DBMR TRIPOD DATA VALIDATION MODEL 27
2.5 PRIMARY INTERVIEWS WITH KEY OPINION LEADERS 30
2.6 MULTIVARIATE MODELLING 31
2.7 MARKET END USER COVERAGE GRID 31
2.8 PRODUCT LIFELINE CURVE 32
2.9 DBMR MARKET POSITION GRID 33
2.10 VENDOR SHARE ANALYSIS 34
2.11 SECONDARY SOURCES 35
2.12 ASSUMPTIONS 35
3 EXECUTIVE SUMMARY 36
4 PREMIUM INSIGHTS 39
4.1 PESTEL ANALYSIS 40
4.2 PORTER FIVE ANALYSIS 41
5 UPDATE ON GERMLINE GENE THERAPY 42
5.1 GERMLINE GENE THERAPY 42
6 NORTH AMERICA GENE THERAPY MARKET, MO 43
6.1 DRIVERS 45
6.1.1 NOVEL APPROACHES TO GENE THERAPY 45
6.1.2 INCREASING PREVALENCE OF GENETIC DISORDERS 45
6.1.3 THE GROWING INVESTMENT BY BIOTECHNOLOGY & PHARMACEUTICAL COMPANIES 46
6.1.4 GROWING DEMAND FOR PERSONALIZED MEDICINE 47
6.2 RESTRAINTS 47
6.2.1 HIGH COST OF GENE THERAPY 47
6.2.2 ETHICAL AND SAFETY CONCERNS 48
6.2.3 COMPLEXITY OF GENE THERAPY 48
6.3 OPPORTUNITIES 49
6.3.1 RISE IN STRATEGIC ACQUISITION AND PARTNERSHIP AMONG ORGANIZATIONS 49
6.3.2 RISING APPROVAL FOR GENE THERAPY PRODUCTS 49
6.4 CHALLENGES 50
6.4.1 STRINGENT REGULATIONS FOR GENE THERAPY PRODUCTS 50
6.4.2 LONG-TERM SAFETY AND EFFICACY 50
7 NORTH AMERICA GENE THERAPY MARKET, BY VECTOR TYPE 51
7.1 OVERVIEW 52
7.2 VIRAL VECTOR 55
7.2.1 ADENOVIRUS 56
7.2.2 RETROVIRUS 56
7.2.3 LENTIVIRUS 56
7.2.4 ADENO-ASSOCIATED VIRUS 56
7.2.5 VACCINIA VIRUS 56
7.2.6 HERPES SIMPLEX VIRUS 56
7.2.7 OTHERS 56
7.3 NON-VIRAL VECTOR 56
7.3.1 LIPOFECTION 57
7.3.2 INJECTION OF NAKED DNA 57
8 NORTH AMERICA GENE THERAPY MARKET, BY METHOD 58
8.1 OVERVIEW 59
8.2 EX-VIVO 62
8.3 IN-VIVO 62
9 NORTH AMERICA GENE THERAPY MARKET, BY APPLICATION 64
9.1 OVERVIEW 65
9.2 ONCOLOGICAL DISORDERS 68
9.3 CARDIOVASCULAR DISEASES 68
9.4 INFECTIOUS DISEASES 69
9.5 RARE DISEASES 70
9.6 NUEROLOGICAL DISORDERS 71
9.7 OTHER DISEASES 72
10 NORTH AMERICA GENE THERAPY MARKET, BY END USER 73
10.1 OVERVIEW 74
10.2 CANCER INSTITUTES 77
10.3 HOSPITALS 77
10.4 RESEARCH INSTITUTES 78
10.5 OTHERS 79
11 NORTH AMERICA GENE THERAPY MARKET, BY REGION 80
11.1 NORTH AMERICA 81
11.1.1 U.S. 87
11.1.2 CANADA 89
11.1.3 MEXICO 91
12 NORTH AMERICA GENE THERAPY MARKET, COMPANY LANDSCAPE 93
12.1 COMPANY SHARE ANALYSIS: NORTH AMERICA 93
13 COMPANY PROFILES 94
13.1 BIOGEN 94
13.1.1 COMPANY SNAPSHOT 94
13.1.2 REVENUE ANALYSIS 94
13.1.3 COMPANY SHARE ANALYSIS 95
13.1.4 SWOT ANALYSIS 95
13.1.5 PRODUCT PORTFOLIO 96
13.1.6 RECENT DEVELOPMENT 96
13.2 KITE PHARMA 97
13.2.1 COMPANY SNAPSHOT 97
13.2.2 REVENUE ANALYSIS 97
13.2.3 COMPANY SHARE ANALYSIS 98
13.2.4 SWOT ANALYSIS 98
13.2.5 PRODUCT PORTFOLIO 99
13.2.6 RECENT DEVELOPMENT 99
13.3 NOVARTIS AG 100
13.3.1 COMPANY SNAPSHOT 100
13.3.2 REVENUE ANALYSIS 100
13.3.3 COMPANY SHARE ANALYSIS 101
13.3.4 SWOT ANALYSIS 101
13.3.5 PRODUCT PORTFOLIO 102
13.3.6 RECENT DEVELOPMENTS 102
13.4 BRISTOL-MYERS SQUIBB COMPANY. 103
13.4.1 COMPANY SNAPSHOT 103
13.4.2 REVENUE ANALYSIS 103
13.4.3 COMPANY SHARE ANALYSIS 104
13.4.4 SWOT ANALYSIS 104
13.4.5 PRODUCT PORTFOLIO 105
13.4.6 RECENT DEVELOPMENT 105
13.5 OXFORD BIOMEDICA 106
13.5.1 COMPANY SNAPSHOT 106
13.5.2 REVENUE ANALYSIS 106
13.5.3 COMPANY SHARE ANALYSIS 107
13.5.4 SWOT ANALYSIS 107
13.5.5 PRODUCT PORTFOLIO 108
13.5.6 RECENT DEVELOPMENTS 108
13.6 AGC BIOLOGICS 109
13.6.1 COMPANY SNAPSHOT 109
13.6.2 PRODUCT PORTFOLIO 109
13.6.3 RECENT DEVELOPMENT 109
13.7 ANGES, INC 110
13.7.1 COMPANY SNAPSHOT 110
13.7.2 REVENUE ANALYSIS 110
13.7.3 PRODUCT PORTFOLIO 111
13.7.4 RECENT DEVELOPMENT 111
13.8 AMGEN INC. 112
13.8.1 COMPANY SNAPSHOT 112
13.8.2 REVENUE ANALYSIS 112
13.8.3 PRODUCT PORTFOLIO 113
13.8.4 RECENT DEVELOPMENT 113
13.9 BLUEBIRD BIO, INC. 114
13.9.1 COMPANY SNAPSHOT 114
13.9.2 PRODUCT PORTFOLIO 114
13.9.3 RECENT DEVELOPMENT 114
13.10 CHIESI FARMACEUTICI S.P.A 115
13.10.1 COMPANY SNAPSHOT 115
13.10.2 REVENUE ANALYSIS 115
13.10.3 PRODUCT PORTFOLIO 116
13.10.4 RECENT DEVELOPMENT 116
13.11 DENDREON PHARMACEUTICALS LLC 117
13.11.1 COMPANY SNAPSHOT 117
13.11.2 PRODUCT PORTFOLIO 117
13.11.3 RECENT DEVELOPMENT 117
13.12 ENZYVANT THERAPEUTICS GMBH 118
13.12.1 COMPANY SNAPSHOT 118
13.12.2 RODUCT PORTFOLIO 118
13.12.3 RECENT DEVELOPMENT 118
13.13 FERRING B.V. 119
13.13.1 COMPANY SNAPSHOT 119
13.13.2 PRODUCT PORTFOLIO 119
13.13.3 RECENT DEVELOPMENT 119
13.14 JANSSEN PHARMACEUTICALS, INC. 120
13.14.1 COMPANY SNAPSHOT 120
13.14.2 PRODUCT PORTFOLIO 120
13.14.3 RECENT DEVELOPMENT 120
13.15 MALLINCKRODT. 121
13.15.1 COMPANY SNAPSHOT 121
13.15.2 REVENUE ANALYSIS 121
13.15.3 PRODUCT PORTFOLIO 122
13.15.4 RECENT DEVELOPMENT 122
13.16 ORCHARD THERAPEUTICS PLC. 123
13.16.1 COMPANY SNAPSHOT 123
13.16.2 REVENUE ANALYSIS 123
13.16.3 PRODUCT PORTFOLIO 124
13.16.4 RECENT DEVELOPMENT 124
13.17 SHANGHAI SUNWAY BIOTECH CO., LTD. 125
13.17.1 COMPANY SNAPSHOT 125
13.17.2 PRODUCT PORTFOLIO 125
13.17.3 RECENT DEVELOPMENT 125
13.18 SIBONO 126
13.18.1 COMPANY SNAPSHOT 126
13.18.2 PRODUCT PORTFOLIO 126
13.18.3 RECENT DEVELOPMENT 126
13.19 SPARK THERAPEUTICS, INC. 127
13.19.1 COMPANY SNAPSHOT 127
13.19.2 PRODUCT PORTFOLIO 127
13.19.3 RECENT DEVELOPMENT 127
13.20 UNIQURE NV. 128
13.20.1 COMPANY SNAPSHOT 128
13.20.2 REVENUE ANALYSIS 128
13.20.3 PRODUCT PORTFOLIO 129
13.20.4 RECENT DEVELOPMENT 129
14 QUESTIONNAIRE 130
15 RELATED REPORTS 133

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