Date Published
November 2015

Page Count 

Additional Info 
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Tables and Figures

Figure 2.1. Gammaretroviral Vector Construction and Packaging 40
Figure 3.1. The Glybera Vector DNA 52
Figure 4.1. Structure of GenSight AAV2/2-CMV-ND4 Vector (GS010) 68
Figure 7.1. CAR T cell 105


Table 2.1: Leading Gene Therapy Vectors 48
Table 3.1. uniQure’s Clinical-Stage Pipeline 56
Table 4.1: Clinical Stage Gene Therapies for Ophthalmological Diseases 62
Table 5.1: Clinical-stage gene therapies for hemophilia B 78
Table 5.2: Clinical-Stage Gene Therapies for Selected Other Rare Diseases 82
Table 6.1: Voyager’s Preclinical Programs 94
Table 7.1: bluebird bio’s Clinical-Stage Gene Therapies 100
Table 7.2: Selected Clinical Programs in CAR T-cell-based Immunotherapy 106
Table 8.1: Companies Involved in Gene Editing Technologies 119
Table 9.1: Gene therapy products likely to reach the market before 2020 155