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Gene Therapy for Mucopolysaccharidosis - A Pipeline Analysis Report

Gene Therapy for Mucopolysaccharidosis - A Pipeline Analysis Report

This pipeline analysis report provides detailed insights into the clinical trials landscape of the gene therapy for mucopolysaccharidosis including molecules at pre-clinical and various other development stages. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations including route of administration

Key questions answered in the report include

  • What are the gene therapy molecules in the various development stages for mucopolysaccharidosis?
  • What are the companies that are currently involved in the development of gene therapy molecules for mucopolysaccharidosis?
  • Insight into discontinued/inactive molecules with appropriate reasoning?
  • What are the major regulatory authorities approving drugs in various regions?
  • Detailed profiling of each active molecule.


  • Executive summary
  • Scope of the report
  • Research methodology
  • Gene therapy for mucopolysacchari doses: An insight
    • Introduction
  • Major regulatory authorities
    • US
      • Table Drug approval process by US FDA
    • Europe
    • Japan
    • Australia
    • China
  • Pipeline landscape
    • Table Pipeline molecules (active) by vendors
    • Table Pipeline landscape
  • Comparative analysis
    • Table Pipeline molecules in different developmental stages 2017
    • Table Percentage of pipeline molecules in different developmental stages 2017
    • Mid-stage molecules (Phase I/II), 2017
      • Table Overview: ABO-101
      • Table Clinical trials description of ABO-101
      • Table Overview: ABO-102
      • Table Clinical trials description of AB0-102
      • Table Overview: SAF-301
      • Table Clinical trials description of SAF-301
      • Table Overview: SAF302
      • Table Overview: SB-913
      • Table Clinical trials description of SB-913
      • Table Overview: SB-318
      • Table Clinical trials description of SB-318
      • Table Overview: EGT-101
      • Table Clinical trials description of EGT-101
    • Early-stage molecules (pre-IND and IND), 2017
      • Table Overview: Autologous ex-vivo lentiviral gene therapy
      • Table Overview: RGX-111
      • Table Overview: RGX-121
    • Pre-clinical stage molecules, 2017
      • Table Overview: EGT-201
      • Table Overview: EGT-301
      • Table Overview: Gene therapy for mucopolysaccharidosis I (Hurler's syndrome)
      • Table Overview: SOBI003
    • Suspended molecule, 2017
      • Table Suspended molecule, 2017
  • Indication analysis
    • Table Analysis by type of indication
  • Therapeutic assessment by RoA
    • Table Assessment by RoA
    • Table Assessment of gene therapy for mucopolysaccharidosis by stage and RoA
  • Key companies
    • Active companies: Category and parameters
      • Table Active companies: Category and parameters 2017
      • Table Segmentation of companies 2017
      • Table Overview: Abeona Therapeutics
      • Table Overview: ESTEVE
      • Table Overview: Immusoft
      • Table Overview: Lysogene
      • Table Overview: Orchard Therapeutics
      • Table Overview: REGENXBIO
      • Table Overview: Sangamo Therapeutics
      • Table Overview: Swedish Orphan Biovitrum
      • Table Overview: uniQure
  • Appendix
    • List of abbreviations
  • Explore Technavio

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