List of Tables
Table 1: Pipeline Products Covered
Table 2: Properties of Standard Gene-Editing Nucleases
Table 3: Registered Clinical Trials of CAR-T Cell Therapies Using Gene-Editing
Table 4: Gene Therapy Clinical Trials Worldwide by Vector
Table 5: Gene Editing Landscape, Vector Differentiation by Target Tissue
Table 6: Gene Editing Landscape, Key Off-Target Effect Studies
Table 7: Pre-existing Immunity to Cas9
Table 8: Characterization of the Rate of Homology Directed Repair in Range of Cell Lines
Table 9: Comparison of Gene Editing and Gene Transfer Approaches
Table 10: Gene Editing Landscape, Pipeline of Targeted Gene Editing Products by Company
Table 11: Luxturna Clinical Studies
Table 12: EDIT-101 – Phase I/II Trial Design and Comparison to Luxturna Efficacy Study
Table 13: Dose Response in CEP290 Gene Editing and CRISPR Expression
Table 14: Gene editing landscape, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), Diagnosed, Prevalent Cases, 2017-2027
Table 15: Key Phase I/II Studies in MPS I
Table 16: Gene editing landscape, Mucopolysaccharidosis II (MPS II) (Hunter Syndrome), Diagnosed, Prevalent Cases, 2017-2027.
Table 17: SB-913 Trial Design
Table 18: SB-913 – Interim Results
Table 19: LYS-SAF302 Phase II/III Trial Design
Table 20: Gene Editing Landscape, MPS Disorders
Table 21: Gene Editing Landscape, Hemophilia Epidemiology and Forecast, 2016-2026.
Table 22: Scale of Hemophilia Severity
Table 23: Phase I/II Hemophilia A Trials
Table 24: BMN-270 Factor VIII Levels at 1.5 Years (High Dose)
Table 25: Gene Therapy Landscape, Phase I/II Clinical Trial Design for Hemophilia A
Table 26: Gene Therapy Landscape, Summary of Key Pipeline Gene Therapies for Hemophilia A
Table 27: Phase I/II Clinical Studies
Table 28: Gene Therapy Landscape, Overview Of Key Pipeline Products in Hemophilia B
Table 29: Gene editing landscape, Thalassemia, Diagnosed, Prevalent Cases, 2017-2027
Table 30: Frequent Mutations Causing SCD
Table 31: Phase I/II Clinical Studies
Table 32: LentiGlobin - Ongoing Phase III Trials in Beta Thalassemia
Table 33: Results of LentiGlobin trials in Thalassemia and Sickle Cell Disease
Table 34: ST-400 Trial Design Compared with LentiGlobin
Table 35: CTX-001 trial design (Left) against ST-400 and LentiGlobin (Right)
Table 36: LentiGlobin (BB-305) may Achieve Blockbuster Status by 2023
Table 37: Gene Therapy Landscape, DMD, Global Prevalence (%)
Table 38: Exondys 51 – Clinical Studies
Table 39: SGT- 001 Study Design
Table 40: Gene Therapy Landscape, Key Patents in the CRISPR Dispute
Table 41: Gene Therapy Landscape, IP Estates of CRISPR Companies
Table 42: Gene Therapy Landscape, ERS Genomics EU Licensing 2004-2018
Table 43: Sangamo Therapeutics Pipeline, September 2018
Table 44: Sangamo Therapeutics Partnerships
Table 45: Sangamo Therapeutics SWOT
Table 46: CRISPR Therapeutics Pipeline
Table 47: CRISPR Therapeutics Pipeline
Table 48: CRISPR Therapeutics SWOT
Table 49: Casebia Pipeline, September 2018
Table 50: Editas Medicine Pipeline, September 2018
Table 51: Editas Medicine Pipeline, September 2018.
Table 52: Editas Medicine SWOT
Table 53: Intellia Therapeutics Pipeline
Table 54: Intellia Therapeutics SWOT
Table 55: Homology Medicines Pipeline
Table 56: Homology Medicines SWOT