Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis

SKU ID :CBR-11697386 | Published Date: 01-Jan-2018 | No. of pages: 79
1 Table of Contents 1 Table of Contents 2 1.1 List of Tables 3 1.2 List of Figures 3 2 Gene Therapy Overview 5 2.1 Types of Gene Therapy 7 2.1.1 Types of Intervention 8 2.1.2 Types of Vector 13 3 Currently Approved Gene Therapies 22 3.1 Glybera (alipogene tiparvovec) 22 3.2 Kynamro (mipomersen) 23 3.3 Macugen (pegaptanib) 24 3.4 Vitravene (fomivirsen) 25 3.5 Gendicine (rAd-p53) 26 3.6 Oncorine (rAd5-H101) 26 3.7 Neovasculgen (Pl-VEGF165) 27 3.8 Exondys 51 (eteplirsen) 28 3.9 Spinraza (nusinersen) 29 3.10 Strimvelis (GSK-2696273) 30 3.11 Kymriah (tisagenlecleucel) 32 3.12 Yescarta (axicabtagene ciloleucel) 33 3.13 Imlygic (talimogene laherparepvec) 34 3.14 Zalmoxis (Allogenic T cells encodng LNGFR and HSV-TK) 36 3.15 Luxturna (voretigene neparvovec) 37 4 Gene Therapy Production Strategies 38 4.1 Production of Viral Vectors 38 4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors 40 4.2 Production of Cell-based Gene Therapies 42 5 Challenges to Gene Therapy Development 44 6 Gene Therapy Pipeline and Emerging Technologies 46 6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development 46 6.2 Gene Therapy Pipeline by Intervention and Vector Type 48 6.3 Pipeline for CAR-T Cell Therapies 51 6.4 Company Positioning 52 6.4.1 Companies by Therapy Area 53 6.4.2 Companies by Stage of Development 54 6.4.3 Companies by Intervention Type 54 6.4.4 Companies by Vector Type 56 6.4.5 Companies Developing CAR-T Cell Therapies by Stage 57 6.5 Early Genome Editing Technologies 57 6.5.1 Zinc Finger Nucleases 58 6.5.2 TALEN 58 6.5.3 CRISPR-Cas9 59 7 Strategic Consolidations 62 7.1 Licensing Deals 62 7.1.1 Licensing Deals by Region Value and Year 62 7.1.2 Licensing Deals by Stage of Development and Value 64 7.1.3 Licensing Deals by Intervention and Vector Type 65 7.2 Co-development deals 66 7.2.1 Co-development Deals by Region, Year and Value 66 7.2.2 Co-development Deals by Stage of Development and Value 68 7.2.3 Co-development Deals by Intervention and Vector Type 69 8 Conclusion 71 9 Appendix 72 9.1 References 72 9.2 About GBI Research 79
1.1 List of Tables Table 1: Gene Therapies, Global, Methods of Gene Silencing, 2017 10 Table 2: Gene Therapies, Global, Types of Viral Vector for Gene Therapy Delivery, 2017 15 Table 3: Gene Therapies, Global, Types of Non-Viral Vector for Gene Therapy Delivery, 2017 19 Table 4: Gene Therapies, Global, Clinical Characteristics of Glybera, 2017 23 Table 5: Gene Therapies, Global, Clinical Characteristics of Kynamro, 2017 24 Table 6: Gene Therapies, Global, Clinical Characteristics of Macugen, 2017 25 Table 7: Gene Therapies, Global, Clinical Characteristics of Vitravene, 2017 25 Table 8: Gene Therapies, Global, Clinical Characteristics of Gendicine, 2017 26 Table 9: Gene Therapies, Global, Clinical Characteristics of Oncorine, 2017 27 Table 10: Gene Therapies, Global, Clinical Characteristics of Neovasculgen, 2017 27 Table 11: Gene Therapies, Global, Clinical Characteristics of Exondys 51, 2017 29 Table 12: Gene Therapies, Global, Clinical Characteristics of Spinraza, 2017 30 Table 13: Gene Therapies, Global, Clinical Characteristics of Strimvelis, 2017 32 Table 14: Gene Therapies, Global, Clinical Characteristics of Kymriah, 2017 33 Table 15: Gene Therapies, Global, Clinical Characteristics of Yescarta, 2017 34 Table 16: Gene Therapies, Global, Clinical Characteristics of Imlygic, 2017 35 Table 17: Gene Therapies, Global, Clinical Characteristics of Zalmoxis, 2017 36 Table 18: Gene Therapies, Global, Clinical Characteristics of Luxturna, 2017 37 Table 19: Gene Therapies, Global, Impurities in the AAV Production Process, 2017 41 Table 20: Gene Therapies, Global, Overall Challenges to Gene Therapy Development, 2017 44 Table 21: Gene Therapies, Global, Comparison of CRISPR-Cas9, ZFNs and TALENs as Gene Editing Platforms, 2017 571.2 List of Figures Figure 1: Gene Therapies, Global, Definition of Gene Therapy, 2017 5 Figure 2: Gene Therapies, Global, Clinical Trials Approved for Gene Therapies, 1989-2012 6 Figure 3: Gene Therapies, Global, Timeline of Key Events in Gene Therapy Research, 1944-2017 7 Figure 4: Gene Therapies, Global, Types of Gene Therapy-based Intervention, 2017 8 Figure 5: Gene Therapies, Global, Types of Vector for Gene Therapy Delivery, 2017 14 Figure 6: Gene Therapies, Global, Specific Types of Vector for Gene Therapy Delivery, 2017 14 Figure 7: Gene Therapies, Global, Production of Viral Vectors for Gene Therapy; Genome Modification, 2017 39 Figure 8: Gene Therapies, Global, Viral Gene Therapy Vector Manufacture; General Production and Purification Scheme, 2017 40 Figure 9: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017 46 Figure 10: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017 47 Figure 11: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Intervention Type, 2017 48 Figure 12: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Therapy Area, 2017 49 Figure 13: Gene Therapies, Global, Gene Therapy Pipeline by Intervention Type and Therapy Area, 2017 50 Figure 14: Gene Therapies, Global, CAR-T Cell Therapies by Stage of development, 2017 51 Figure 15: Gene Therapies, Global, CAR-T Cell Therapies by Molecular Target, 2017 51 Figure 16: Gene Therapies, Global, Gene Therapy Pipeline Companies by Type and Level of Diversification, 2017 52 Figure 17: Gene Therapies, Global, Gene Therapy Pipeline by Company and Therapy Area, 2017 53 Figure 18: Gene Therapies, Global, Gene Therapy Pipeline by Company and Stage of Development, 2017 54 Figure 19: Gene Therapies, Global, Gene Therapy Pipeline by Company and Intervention Type, 2017 55 Figure 20: Gene Therapies, Global, Gene Therapy Pipeline by Company and Vector Type, 2017 56 Figure 21: Gene Therapies, Global, CAR-T Cell Therapy Pipeline by Company and Stage of development, 2017 57 Figure 22: Gene Therapies, Global, Licensing Deals by Year, 2006-2017 63 Figure 23: Gene Therapies, Global, Licensing Deals by Stage of Development and Value, 2006-2017 64 Figure 24: Gene Therapies, Global, Licensing Deals by Vector type, 2006-2017 65 Figure 25: Gene Therapies, Global, Licensing Deals by Intervention type, 2006-2017 66 Figure 26: Gene Therapies, Global, Co-Development Deals by Year, 2006-2017 67 Figure 27: Gene Therapies, Global,Co-Development Deals by Year and Value, 2006-2017 68 Figure 28: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017 69 Figure 29: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017 70
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