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Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018

Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018

Summary


MPS I is caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 18 products in development for this indication.

MPS II is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 15 products in development for this indication.

MPS III is caused by an absence or malfunctioning of GAGs. Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). There are 18 products in development for this indication.

Companies operating in the mucopolysaccharidosis disorders pipeline space include ArmaGen, Sangamo and AngioChem.

This report Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018 provides an overview of the pipeline landscape for mucopolysaccharidosis disorders, a group of inherited lysosomal storage disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for mucopolysaccharidosis I (MPS I) (Hurler syndrome), mucopolysaccharidosis II (MPS II) (Hunter syndrome) and mucopolysaccharidosis III (MPS III) (Sanfilippo syndrome), and features dormant and discontinued products.

Scope

  • Which companies are the most active within each pipeline?
  • Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
  • To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
  • What are the most important R&D milestones and data publications to have happened in this disease area?
Reasons to buy
  • Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
  • Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication and a comprehensive picture of recent updates and milestones for each
  • Analyze the companies, institutions and universities currently operating in the pipeline and the products being fielded by each of these
  • Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration


  • GBI Research Report Guidance
  • Executive Summary
  • Introduction
    • Mucopolysaccharidosis Disorders Report Coverage
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Overview
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview
  • Therapeutics Development
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Pipeline Overview
        • Table Figure 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Pipeline by Companies
        • Table Figure 2: Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Pipeline by Universities/Institutes
        • Table Figure 3: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Products under Development by Companies
        • Table Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Products under Development by Universities/Institutes
        • Table Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Pipeline Overview
        • Table Figure 4: Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Pipeline by Companies
        • Table Figure 5: Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Pipeline by Universities/Institutes
        • Table Figure 6: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Products under Development by Companies
        • Table Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Products under Development by Universities/Institutes
        • Table Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Pipeline Overview
        • Table Figure 7: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Pipeline by Companies
        • Table Figure 8: Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Products under Development by Companies
        • Table Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
  • Therapeutics Assessment
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Assessment by Target
        • Table Figure 9: Number of Products by Stage and Targets Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products by Stage and Target Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Assessment by Mechanism of Action
        • Table Figure 10: Number of Products by Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Figure 11: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Assessment by Route of Administration
        • Table Figure 12: Number of Products by Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Figure 13: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products by Stage and Route of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Assessment by Molecule Type
        • Table Figure 14: Number of Products by Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Figure 15: Number of Products by Stage and Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
        • Table Number of Products by Stage and Molecule Type Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Assessment by Target
        • Table Figure 16: Number of Products by Stage and Targets Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products by Stage and Target Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Assessment by Mechanism of Action
        • Table Figure 17: Number of Products by Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Figure 18: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Assessment by Route of Administration
        • Table Figure 19: Number of Products by Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Figure 20: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products by Stage and Route of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Assessment by Molecule Type
        • Table Figure 21: Number of Products by Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Figure 22: Number of Products by Stage and Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
        • Table Number of Products by Stage and Molecule Type Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Assessment by Target
        • Table Figure 23: Number of Products by Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Figure 24: Number of Products by Stage and Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products by Stage and Target Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Assessment by Mechanism of Action
        • Table Figure 25: Number of Products by Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Figure 26: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Assessment by Route of Administration
        • Table Figure 27: Number of Products by Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Figure 28: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products by Stage and Route of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Assessment by Molecule Type
        • Table Figure 29: Number of Products by Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Figure 30: Number of Products by Stage and Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
        • Table Number of Products by Stage and Molecule Type Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
  • Companies Involved in Therapeutics Development
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • AngioChem Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc
      • ArmaGen Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Inc
      • CRISPR Therapeutics
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by CRISPR Therapeutics
      • Eloxx Pharmaceuticals Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Eloxx Pharmaceuticals Inc
      • Immusoft Corp
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Immusoft Corp
      • JCR Pharmaceuticals Co Ltd
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd
      • OPKO Health Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health Inc
      • RegenxBio Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by RegenxBio Inc
      • Sangamo Therapeutics Inc
        • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Sangamo Therapeutics Inc
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • AngioChem Inc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by AngioChem Inc
      • ArmaGen Inc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by ArmaGen Inc
      • Denali Therapeutics Inc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Denali Therapeutics Inc
      • GC Pharma
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by GC Pharma
      • JCR Pharmaceuticals Co Ltd
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd
      • Laboratorios Del Dr Esteve SA
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA
      • RegenxBio Inc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by RegenxBio Inc
      • Sangamo Therapeutics Inc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Sangamo Therapeutics Inc
      • Shire Plc
        • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Shire Plc
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Abeona Therapeutics Inc
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Abeona Therapeutics Inc
      • ArmaGen Inc
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by ArmaGen Inc Product
      • Axcentua Pharmaceuticals AB
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Axcentua Pharmaceuticals AB
      • BioMarin Pharmaceutical Inc
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by BioMarin Pharmaceutical Inc
      • Laboratorios Del Dr Esteve SA
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA
      • Lysogene SAS
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Lysogene SAS
      • Swedish Orphan Biovitrum AB
        • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Swedish Orphan Biovitrum AB
  • Dormant Projects
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Dormant Projects
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects
  • Discontinued Products
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Table Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Discontinued Products
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Table Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Discontinued Products
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Table Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Discontinued Products
  • Product Development Milestones
    • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)
      • Featured News & Press Releases
    • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome)
      • Featured News & Press Releases
    • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
      • Featured News & Press Releases
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

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