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Sickle Cell Disease Drug Development Pipeline Review, 2017

Sickle Cell Disease Drug Development Pipeline Review, 2017

Summary

Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. There are a total of 62 products in development for this indication, by 45 companies and nine academic institutions.

Key companies operating in this pipeline space include Acceleron Pharma, Epidestiny, Errant Gene Therapeutics, Gamida Cell, Global Blood Therapeutics, Hillhurst Biopharmaceuticals, Novartis and Regenacy Pharmaceuticals. Hemoglobin and a range of histone deacetylases are the most common molecular targets being studied in sickle cell anemia.

Vaso-occlusive crisis associated with sickle cell disease is a painful episode that occurs in people who have sickle cell anemia. Symptoms include fever, pain, fatigue, abdominal pain and swelling, headaches, shortness of breath, dehydration and infection. There are a total of 10 products in development for this indication, by nine companies.

This report provides an overview of the Sickle Cell Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for sickle cell anemia and vaso-occlusive crisis associated with sickle cell disease, and features dormant and discontinued projects.

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
    • Sickle Cell Disease Report Coverage
    • Sickle Cell Disease - Overview
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Overview
  • Therapeutics Development
    • Sickle Cell Disease
      • Pipeline Overview
        • Table Figure 1: Number of Products under Development for Sickle Cell Disease
        • Table Number of Products under Development for Sickle Cell Disease
      • Pipeline by Companies
        • Table Figure 2: Number of Products under Development by Companies, Sickle Cell Disease
        • Table Number of Products under Development by Companies, Sickle Cell Disease
      • Pipeline by Universities/Institutes
        • Table Figure 3: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
        • Table Number of Products under Development by Universities/Institutes, Sickle Cell Disease
      • Products under Development by Companies
        • Table Products under Development by Companies, Sickle Cell Disease
      • Products under Development by Universities/Institutes
        • Table Products under Development by Universities/Institutes, Sickle Cell Disease
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Pipeline Overview
        • Table Figure 4: Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Pipeline by Companies
        • Table Figure 5: Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Products under Development by Companies
        • Table Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
  • Therapeutics Assessment
    • Sickle Cell Disease
      • Assessment by Target
        • Table Figure 6: Number of Products by Top 10 Targets, Sickle Cell Disease
        • Table Figure 7: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease
        • Table Number of Products by Stage and Target, Sickle Cell Disease
      • Assessment by Mechanism of Action
        • Table Figure 8: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease
        • Table Figure 9: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease
        • Table Number of Products by Stage and Mechanism of Action, Sickle Cell Disease
      • Assessment by Route of Administration
        • Table Figure 10: Number of Products by Routes of Administration, Sickle Cell Disease
        • Table Figure 11: Number of Products by Stage and Routes of Administration, Sickle Cell Disease
        • Table Number of Products by Stage and Route of Administration, Sickle Cell Disease
      • Assessment by Molecule Type
        • Table Figure 12: Number of Products by Top 10 Molecule Types, Sickle Cell Disease
        • Table Figure 13: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease
        • Table Number of Products by Stage and Molecule Type, Sickle Cell Disease
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Assessment by Target
        • Table Figure 14: Number of Products by Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Figure 15: Number of Products by Stage and Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products by Stage and Target, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Assessment by Mechanism of Action
        • Table Figure 16: Number of Products by Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Figure 17: Number of Products by Stage and Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products by Stage and Mechanism of Action, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Assessment by Route of Administration
        • Table Figure 18: Number of Products by Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Figure 19: Number of Products by Stage and Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products by Stage and Route of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Assessment by Molecule Type
        • Table Figure 20: Number of Products by Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Figure 21: Number of Products by Stage and Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
        • Table Number of Products by Stage and Molecule Type, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
  • Companies Involved in Therapeutics Development
    • Sickle Cell Disease
      • Acceleron Pharma Inc
        • Table Sickle Cell Disease - Pipeline by Acceleron Pharma Inc
      • Addmedica SAS
        • Table Sickle Cell Disease - Pipeline by Addmedica SAS
      • Angiocrine Bioscience Inc
        • Table Sickle Cell Disease - Pipeline by Angiocrine Bioscience Inc
      • Annexin Pharmaceuticals AB
        • Table Sickle Cell Disease - Pipeline by Annexin Pharmaceuticals AB
      • ArQule Inc
        • Table Sickle Cell Disease - Pipeline by ArQule Inc
      • Bio Products Laboratory Ltd
        • Table Sickle Cell Disease - Pipeline by Bio Products Laboratory Ltd
      • Bioverativ Inc
        • Table Sickle Cell Disease - Pipeline by Bioverativ Inc
      • bluebird bio Inc
        • Table Sickle Cell Disease - Pipeline by bluebird bio Inc
      • Bristol-Myers Squibb Co
        • Table Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Co
      • Calimmune Inc
        • Table Sickle Cell Disease - Pipeline by Calimmune Inc
      • CRISPR Therapeutics
        • Table Sickle Cell Disease - Pipeline by CRISPR Therapeutics
      • CSL Ltd
        • Table Sickle Cell Disease - Pipeline by CSL Ltd
      • Daiichi Sankyo Co Ltd
        • Table Sickle Cell Disease - Pipeline by Daiichi Sankyo Co Ltd
      • Editas Medicine Inc
        • Table Sickle Cell Disease - Pipeline by Editas Medicine Inc
      • Errant Gene Therapeutics LLC
        • Table Sickle Cell Disease - Pipeline by Errant Gene Therapeutics LLC
      • Gamida Cell Ltd
        • Table Sickle Cell Disease - Pipeline by Gamida Cell Ltd
      • Genethon SA
        • Table Sickle Cell Disease - Pipeline by Genethon SA
      • Gilead Sciences Inc
        • Table Sickle Cell Disease - Pipeline by Gilead Sciences Inc
      • Global Blood Therapeutics Inc
        • Table Sickle Cell Disease - Pipeline by Global Blood Therapeutics Inc
      • Incyte Corp
        • Table Sickle Cell Disease - Pipeline by Incyte Corp
      • Ironwood Pharmaceuticals Inc
        • Table Sickle Cell Disease - Pipeline by Ironwood Pharmaceuticals Inc
      • La Jolla Pharmaceutical Company
        • Table Sickle Cell Disease - Pipeline by La Jolla Pharmaceutical Company
      • MaxCyte Inc
        • Table Sickle Cell Disease - Pipeline by MaxCyte Inc
      • Merck & Co Inc
        • Table Sickle Cell Disease - Pipeline by Merck & Co Inc
      • Morphogenesis Inc
        • Table Sickle Cell Disease - Pipeline by Morphogenesis Inc
      • NKT Therapeutics Inc
        • Table Sickle Cell Disease - Pipeline by NKT Therapeutics Inc
      • Novartis AG
        • Table Sickle Cell Disease - Pipeline by Novartis AG
      • Orphagen Pharmaceuticals Inc
        • Table Sickle Cell Disease - Pipeline by Orphagen Pharmaceuticals Inc
      • Prolong Pharmaceuticals LLC
        • Table Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals LLC
      • Protagonist Therapeutics Inc
        • Table Sickle Cell Disease - Pipeline by Protagonist Therapeutics Inc
      • ReveraGen BioPharma Inc
        • Table Sickle Cell Disease - Pipeline by ReveraGen BioPharma Inc
      • Sancilio & Company Inc
        • Table Sickle Cell Disease - Pipeline by Sancilio & Company Inc
      • Sangamo Therapeutics Inc
        • Table Sickle Cell Disease - Pipeline by Sangamo Therapeutics Inc
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • AstraZeneca Plc
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by AstraZeneca Plc
      • Bristol-Myers Squibb Co
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Co
      • Gilead Sciences Inc
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Gilead Sciences Inc
      • Modus Therapeutics Holding AB
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Modus Therapeutics Holding AB
      • Novartis AG
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Novartis AG
      • NuvOx Pharma LLC
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by NuvOx Pharma LLC
      • Pfizer Inc
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Pfizer Inc
      • Prolong Pharmaceuticals LLC
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals LLC
      • Seattle Genetics Inc
        • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline by Seattle Genetics Inc
  • Dormant Projects
    • Sickle Cell Disease
      • Table Sickle Cell Disease - Dormant Projects
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Dormant Projects
  • Discontinued Products
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Table Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Discontinued Products
  • Product Development Milestones
    • Sickle Cell Disease
      • Featured News & Press Releases
    • Vaso-Occlusive Crisis Associated With Sickle Cell Disease
      • Featured News & Press Releases
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

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