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Emerging Gene Therapies – Trends within the Technological, Clinical, Regulatory and Competitive Landscape

  • Published: Jan-2019
  • Report Code: GBI095CBR
  • Report Format: pdf


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

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