United States Gene Therapy Sales Market Report 2021

1 Gene Therapy Overview 1

1.1 Product Overview and Scope of Gene Therapy 1

1.2 How does Gene Therapy work 1

1.3 Classification of Gene Therapy 3

1.4 Applications of Gene Therapy 6

1.5 USA Market Size (Value and Volume) of Gene Therapy (2011-2021) 7

1.5.1 USA Gene Therapy Investment (2011-2021) 7

1.5.2 USA Gene Therapy Investment and Growth Rate (2011-2021) 8

2 USA Gene Therapy Competition by Manufacturers 10

2.1 USA Gene Therapy Investment Analysis of Key Manufacturers (2015 and 2016) 10

2.2 USA Gene Therapy Investment Market Share of Key Manufacturers (2015 and 2016) 11

3 USA Gene Therapy by Type 13

3.1 USA Gene Therapy Investment Analysis by Type (2011-2021) 13

3.2 USA Gene Therapy Investment and Growth Rate by Type (2011-2021) 15

4 USA Gene Therapy by Application 17

4.1 USA Gene Therapy Investment Analysis by Application (2011-2021) 17

4.2 USA Gene Therapy Investment and Growth Rate by Application (2011-2021) 19

5 USA Gene Therapy Manufacturers Analysis 24

5.1 Bluebird Bio 24

5.1.1 Company Basic Information, Manufacturing Base and Competitors 24

5.1.2 Gene Therapy Product Type and Technology 25

5.1.3 Gene Therapy Sales, Revenue, Price of Bluebird Bio (2015 and 2016) 27

5.2 Sangamo 27

5.2.1 Company Basic Information, Manufacturing Base and Competitors 27

5.2.2 Gene Therapy Product Type and Technology 29

5.2.3 Gene Therapy Sales, Revenue, Price of Sangamo (2015 and 2016) 31

5.3 Spark Therapeutics 31

5.3.1 Company Basic Information, Manufacturing Base and Competitors 31

5.3.2 Gene Therapy Product Type and Technology 32

5.3.3 Gene Therapy Sales, Revenue, Price of Spark Therapeutics (2015 and 2016) 35

5.4 Dimension Therapeutics 36

5.4.1 Company Basic Information, Manufacturing Base and Competitors 36

5.4.2 Gene Therapy Product Type and Technology 37

5.4.3 Gene Therapy Sales, Revenue, Price of Dimension Therapeutics (2015 and 2016) 38

5.5 Avalanche Bio 38

5.5.1 Company Basic Information, Manufacturing Base and Competitors 38

5.5.2 Gene Therapy Product Type and Technology 40

5.5.3 Gene Therapy Sales, Revenue, Price of Avalanche Bio (2015 and 2016) 40

5.6 Celladon 41

5.6.1 Company Profile 41

5.6.2 Gene Technology 42

5.6.3 Investment, Market Share and Growth Rate 42

5.7 Vical 43

5.7.1 Company Profile 43

5.7.2 Gene Technology 44

5.7.3 Investment, Market Share and Growth Rate 46

5.8 Advantagene 46

5.8.1 Company Profile 46

5.8.2 Gene Technology 48

5.8.3 Investment, Market Share and Growth Rate 51

6 Gene Therapy Technology and Development Trend 52

6.1 Gene Therapy Technology Analysis 52

6.1.1 Gene Augmentation Therapy 56

6.1.2 Gene Inhibition Therapy 58

6.1.3 Killing of Specific Cells 60

6.2 Gene Therapy Technology Development Trend 61

7 Global Gene Therapy Trials Analysis 63

7.1 Global Gene Therapy Trials Analysis by Region 63

7.2 Global Gene Therapy Trials Analysis by Country 64

7.3 Global Gene Therapy Trials Analysis by Indications 66

7.4 Global Gene Therapy Trials Analysis by Gene Type 68

7.5 Global Gene Therapy Trials Analysis by Phases 69

7.6 Global Number of Gene Therapy Trials Approved 1989-2016.02 71

8 Research Findings and Conclusion 72

Disclosure Section 73

Research Methodology 73

Data Source 75

China Disclaimer 75

Figure Picture of Gene Therapy 1

Figure Picture of Viruses Work 2

Figure Picture of the Process of Inserting the Vector in Gene Therapy 2

Figure Classification of Gene Therapy 3

Figure USA Gene Therapy Investment by Types in 2015 5

Figure Applications of Gene Therapy 6

Figure USA Gene Therapy Investment by Applications in 2015 7

Table 2011-2021 USA Gene Therapy Investment (K USD) 7

Figure 2011-2016 USA Gene Therapy Investment (K USD) and Growth Rate 8

Figure 2016-2021 USA Gene Therapy Investment (K USD) and Growth Rate 9

Table USA Gene Therapy Investment of Key Manufacturers (2015 and 2016) 10

Table USA Gene Therapy Investment Market Share by Manufacturers (2015 and 2016) 10

Figure 2015 Gene Therapy Investment Market Share by Manufacturers 11

Figure 2016 Gene Therapy Investment Market Share by Manufacturers 12

Table USA Gene Therapy Investment by Type (2011-2021) 13

Table USA Gene Therapy Investment Market Share by Type (2011-2021) 13

Figure Investment Market Share of Gene Therapy by Type (2011-2021) 14

Figure 2011-2016 USA Gene Therapy Ex Vivo Investment and Growth Rate 15

Figure 2016-2021 USA Gene Therapy Ex Vivo Investment and Growth Rate 15

Figure 2011-2016 USA Gene Therapy In Vivo Investment and Growth Rate 16

Figure 2016-2021 USA Gene Therapy In Vivo Investment and Growth Rate 16

Table USA Gene Therapy Investment by Application (2011-2021) 17

Table USA Gene Therapy Investment Market Share by Application (2011-2021) 17

Figure Investment Market Share of Gene Therapy by Application (2011-2021) 18

Figure 2011-2016 USA Gene Therapy Cancer Diseases Investment and Growth Rate 19

Figure 2016-2021 USA Gene Therapy Cancer Diseases Investment and Growth Rate 20

Figure 2011-2016 USA Gene Therapy Monogenic Diseases Investment and Growth Rate 20

Figure 2016-2021 USA Gene Therapy Monogenic Diseases Investment and Growth Rate 21

Figure 2011-2016 USA Gene Therapy Infectious Diseases Investment and Growth Rate 21

Figure 2016-2021 USA Gene Therapy Infectious Diseases Investment and Growth Rate 22

Figure 2011-2016 USA Gene Therapy Cardiovascular Diseases Investment and Growth Rate 22

Figure 2016-2021 USA Gene Therapy Cardiovascular Diseases Investment and Growth Rate 23

Table Bluebird Bio Basic Information List 24

Figure Program Pipeline 25

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Bluebird Bio (2015 and 2016) 27

Table Sangamo Basic Information List 27

Figure Product Pipeline 29

Figure Zinc Finger Proteins (ZFPs) 30

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Sangamo (2015 and 2016) 31

Table Spark Therapeutics Basic Information List 31

Figure Product Pipeline 32

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Spark Therapeutics (2015 and 2016) 35

Table Dimension Therapeutics Basic Information List 36

Figure Product Pipeline 37

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Dimension Therapeutics (2015 and 2016) 38

Table Avalanche Bio Basic Information List 38

Figure Rare Disease Programs Pipeline 40

Figure Ophthalmology Programs Pipeline 40

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Avalanche Bio (2015 and 2016) 40

Table Celladon Information List 41

Figure Clinical Trials Program of Celladon 42

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Celladon (2015 and 2016) 42

Table Vical Information List 43

Figure DNA Vaccine Concept of Vical 45

Figure Gene Therapy Clinical Trials Program of Vical 46

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Vical (2015 and 2016) 46

Table Advantagene Information List 47

Figure Gene Mediated Cytotoxic Immunotherapy of Advantagene 50

Figure Gene Therapy Clinical Trials Program of Advantagene 51

Table Gene Therapy Investment (K USD) , Market Share and Growth Rate of Advantagene (2015 and 2016) 51

Table Characteristic Diseases Caused by Gene Mutations that Code these Products 53

Table Human Gene Therapy Trials Conducted 54

Table Challenges of Gene Therapy 55

Table Key Facts of Gene Augmentation Therapy 56

Figure Gene Augmentation Therapy 58

Table Key Facts of Gene Augmentation Therapy 58

Figure Gene Inhibition Therapy 59

Table Key Facts of Killing of Specific Cells 60

Figure Killing of Specific Cells 61

Table Gene Therapy Trials Analysis by Region 63

Figure Gene Therapy Trials Distribution by Region 64

Table Gene Therapy Trials Analysis by Country 64

Figure Gene Therapy Trials Distribution by Country 66

Table Gene Therapy Trials Analysis by Indications 66

Figure Gene Therapy Trials Distribution by Indications 67

Table Gene Therapy Trials Analysis by Gene Type 68

Figure Gene Therapy Trials Distribution by Gene Type 69

Table Gene Therapy Trials Analysis by Phases 69

Figure Gene Therapy Trials Distribution by Phases 70

Figure Global Number of Gene Therapy Trials Approved 1989-2016.02 71