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Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

Summary


Thalassemia refers to a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include paleness, frequent infections and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant. There are 40 products in development for this indication.

Hemophilia B is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Symptoms include bleeding into joints and associated pain and swelling, bruising, nosebleeds and prolonged bleeding from injuries and surgery. Treatment includes replacing the defective clotting factor. There are 42 products in development for this indication.

Sickle cell anemia is a genetic 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. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant. There are 68 products in development for this indication.

Molecular targets acted on by products in development for hereditary hematological disorders include coagulation factors, histone deacetylases and protein kinases. Companies operating in this pipeline space include Sangamo Therapeutics, Gamida Cell and Bluebird Bio.

This report “Hereditary Hematological Disorders Drug Development Pipeline Review, 2018” provides an overview of the pipeline landscape for hereditary hematological disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for thalassemia, hemophilia B and sickle cell anemia, 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
    • Hereditary Hematological Disorders Report Coverage
    • Thalassemia – Overview
    • Hemophilia B – Overview
    • Sickle Cell Disease – Overview
  • Therapeutics Development
    • Thalassemia
      • Pipeline Overview
        • Table Figure 1: Number of Products under Development for Thalassemia
        • Table Number of Products under Development for Thalassemia
      • Pipeline by Companies
        • Table Figure 2: Number of Products under Development by Companies, Thalassemia
        • Table Number of Products under Development by Companies, Thalassemia
      • Pipeline by Universities/Institutes
        • Table Figure 3: Number of Products under Development by Universities/Institutes, Thalassemia
        • Table Number of Products under Development by Universities/Institutes, Thalassemia
      • Products under Development by Companies
        • Table Products under Development by Companies, Thalassemia
      • Products under Development by Universities/Institutes
        • Table Products under Development by Universities/Institutes, Thalassemia
    • Hemophilia B – Therapeutics Development
      • Pipeline Overview
        • Table Figure 4: Number of Products under Development for Hemophilia B
        • Table Number of Products under Development for Hemophilia B
      • Pipeline by Companies
        • Table Figure 5: Number of Products under Development by Companies, Hemophilia B
        • Table Number of Products under Development by Companies, Hemophilia B
      • Pipeline by Universities/Institutes
        • Table Figure 6: Number of Products under Development by Universities/Institutes, Hemophilia B
        • Table Number of Products under Development by Universities/Institutes, Hemophilia B
      • Products under Development by Companies
        • Table Products under Development by Companies, Hemophilia B
      • Products under Development by Universities/Institutes
        • Table Products under Development by Universities/Institutes, Hemophilia B
    • Sickle Cell Disease – Therapeutics Development
      • Pipeline Overview
        • Table Figure 7: Number of Products under Development for Sickle Cell Disease
        • Table Number of Products under Development for Sickle Cell Disease
      • Pipeline by Companies
        • Table Figure 8: 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 9: 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
  • Therapeutics Assessment
    • Thalassemia – Therapeutics Assessment
      • Assessment by Target
        • Table Figure 10: Number of Products by Top 10 Targets, Thalassemia
        • Table Figure 11: Number of Products by Stage and Top 10 Targets, Thalassemia
        • Table Number of Products by Stage and Target, Thalassemia
      • Assessment by Mechanism of Action
        • Table Figure 12: Number of Products by Top 10 Mechanism of Actions, Thalassemia
        • Table Figure 13: Number of Products by Stage and Top 10 Mechanism of Actions, Thalassemia
        • Table Number of Products by Stage and Mechanism of Action, Thalassemia
      • Assessment by Route of Administration
        • Table Figure 14: Number of Products by Routes of Administration, Thalassemia
        • Table Figure 15: Number of Products by Stage and Routes of Administration, Thalassemia
        • Table Number of Products by Stage and Route of Administration, Thalassemia
      • Assessment by Molecule Type
        • Table Figure 16: Number of Products by Molecule Types, Thalassemia
        • Table Figure 17: Number of Products by Stage and Molecule Types, Thalassemia
        • Table Number of Products by Stage and Molecule Type, Thalassemia
    • Hemophilia B – Therapeutics Assessment
      • Assessment by Target
        • Table Figure 18: Number of Products by Targets, Hemophilia B
        • Table Figure 19: Number of Products by Stage and Targets, Hemophilia B
        • Table Number of Products by Stage and Target, Hemophilia B
      • Assessment by Mechanism of Action
        • Table Figure 20: Number of Products by Mechanism of Actions, Hemophilia B
        • Table Figure 21: Number of Products by Stage and Mechanism of Actions, Hemophilia B
        • Table Number of Products by Stage and Mechanism of Action, Hemophilia B
      • Assessment by Route of Administration
        • Table Figure 22: Number of Products by Routes of Administration, Hemophilia B
        • Table Figure 23: Number of Products by Stage and Routes of Administration, Hemophilia B
        • Table Number of Products by Stage and Route of Administration, Hemophilia B
      • Assessment by Molecule Type
        • Table Figure 24: Number of Products by Molecule Types, Hemophilia B
        • Table Figure 25: Number of Products by Stage and Molecule Types, Hemophilia B
        • Table Number of Products by Stage and Molecule Type, Hemophilia B
    • Sickle Cell Disease – Therapeutics Assessment
      • Assessment by Target
        • Table Figure 26: Number of Products by Top 10 Targets, Sickle Cell Disease
        • Table Figure 27: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease
        • Table Number of Products by Stage and Target, H2 2018, Sickle Cell Disease
      • Assessment by Mechanism of Action
        • Table Figure 28: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease
        • Table Figure 29: 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 30: Number of Products by Routes of Administration, Sickle Cell Disease
        • Table Figure 31: 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 32: Number of Products by Top 10 Molecule Types, Sickle Cell Disease
        • Table Figure 33: 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
  • Companies Involved in Therapeutics Development
    • Thalassemia – Companies Involved in Therapeutics Development
      • Acceleron Pharma Inc
        • Table Thalassemia – Pipeline by Acceleron Pharma Inc
      • Agios Pharmaceuticals Inc
        • Table Thalassemia – Pipeline by Agios Pharmaceuticals Inc
      • bluebird bio Inc
        • Table Thalassemia – Pipeline by bluebird bio Inc
      • Cadila Healthcare Ltd
        • Table Thalassemia – Pipeline by Cadila Healthcare Ltd,
      • Calimmune Inc
        • Table Thalassemia – Pipeline by Calimmune Inc
      • CRISPR Therapeutics
        • Table Thalassemia – Pipeline by CRISPR Therapeutics
      • Editas Medicine Inc
        • Table Thalassemia – Pipeline by Editas Medicine Inc
      • Errant Gene Therapeutics LLC
        • Table Thalassemia – Pipeline by Errant Gene Therapeutics LLC
      • Gamida Cell Ltd
        • Table Thalassemia – Pipeline by Gamida Cell Ltd
      • Gilead Sciences Inc
        • Table Thalassemia – Pipeline by Gilead Sciences Inc
      • Incyte Corp
        • Table Thalassemia – Pipeline by Incyte Corp
      • Ionis Pharmaceuticals Inc
        • Table Thalassemia – Pipeline by Ionis Pharmaceuticals Inc
      • Kiadis Pharma NV
        • Table Thalassemia – Pipeline by Kiadis Pharma NV
      • La Jolla Pharmaceutical Company
        • Table Thalassemia – Pipeline by La Jolla Pharmaceutical Company
      • Merck & Co Inc
        • Table Thalassemia – Pipeline by Merck & Co Inc
      • Poseida Therapeutics Inc
        • Table Thalassemia – Pipeline by Poseida Therapeutics Inc
      • Protagonist Therapeutics Inc
        • Table Thalassemia – Pipeline by Protagonist Therapeutics Inc
      • Sangamo Therapeutics Inc
        • Table Thalassemia – Pipeline by Sangamo Therapeutics Inc
      • Silence Therapeutics Plc
        • Table Thalassemia – Pipeline by Silence Therapeutics Plc
      • Vifor Pharma AG
        • Table Thalassemia – Pipeline by Vifor Pharma AG
    • Hemophilia B – Companies Involved in Therapeutics Development
      • Amarna Therapeutics BV
        • Table Hemophilia B – Pipeline by Amarna Therapeutics BV
      • Bayer AG
        • Table Hemophilia B – Pipeline by Bayer AG
      • Bioverativ Inc
        • Table Hemophilia B – Pipeline by Bioverativ Inc
      • Catalyst Biosciences Inc
        • Table Hemophilia B – Pipeline by Catalyst Biosciences Inc
      • China Biologic Products Inc
        • Table Hemophilia B – Pipeline by China Biologic Products Inc
      • CSL Ltd
        • Table Hemophilia B – Pipeline by CSL Ltd
      • Expression Therapeutics LLC
        • Table Hemophilia B – Pipeline by Expression Therapeutics LLC
      • GC Pharma
        • Table Hemophilia B – Pipeline by GC Pharma
      • Genethon SA
        • Table Hemophilia B – Pipeline by Genethon SA
      • LFB SA
        • Table Hemophilia B – Pipeline by LFB SA
      • Logicbio Therapeutics Inc
        • Table Hemophilia B – Pipeline by Logicbio Therapeutics Inc
      • Novo Nordisk AS
        • Table Hemophilia B – Pipeline by Novo Nordisk AS
      • OPKO Biologics Ltd
        • Table Hemophilia B – Pipeline by OPKO Biologics Ltd
      • Pfizer Inc
        • Table Hemophilia B – Pipeline by Pfizer Inc
      • Pharming Group NV
        • Table Hemophilia B – Pipeline by Pharming Group NV
      • Promethera Biosciences SA
        • Table Hemophilia B – Pipeline by Promethera Biosciences SA
      • RegenxBio Inc
        • Table Hemophilia B – Pipeline by RegenxBio Inc
      • Sangamo Therapeutics Inc
        • Table Hemophilia B – Pipeline by Sangamo Therapeutics Inc
      • Sanofi
        • Table Hemophilia B – Pipeline by Sanofi
      • Shire Plc
        • Table Hemophilia B – Pipeline by Shire Plc
      • Spark Therapeutics Inc
        • Table Hemophilia B – Pipeline by Spark Therapeutics Inc
      • UniQure NV
        • Table Hemophilia B – Pipeline by UniQure NV
    • Sickle Cell Disease – Companies Involved in Therapeutics Development
      • 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
      • Cell Source Inc
        • Table Sickle Cell Disease – Pipeline by Cell Source Inc
      • CRISPR Therapeutics
        • Table Sickle Cell Disease – Pipeline by CRISPR Therapeutics
      • CSL Ltd
        • Table Sickle Cell Disease – Pipeline by CSL Ltd
      • Editas Medicine Inc
        • Table Sickle Cell Disease – Pipeline by Editas Medicine Inc
      • Epizyme Inc
        • Table Sickle Cell Disease – Pipeline by Epizyme Inc
      • Errant Gene Therapeutics LLC
        • Table Sickle Cell Disease – Pipeline by Errant Gene Therapeutics LLC
      • Fulcrum Therapeutics Inc
        • Table Sickle Cell Disease – Pipeline by Fulcrum Therapeutics Inc
      • 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
      • Homology Medicines, Inc.
        • Table Sickle Cell Disease – Pipeline by Homology Medicines, Inc
      • Incyte Corp
        • Table Sickle Cell Disease – Pipeline by Incyte Corp
      • Intellia Therapeutics Inc
        • Table Sickle Cell Disease – Pipeline by Intellia Therapeutics Inc
      • 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
      • MimeTech Srl
        • Table Sickle Cell Disease – Pipeline by MimeTech Srl
      • 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
      • Oryzon Genomics SA
        • Table Sickle Cell Disease – Pipeline by Oryzon Genomics SA
      • Pfizer Inc
        • Table Sickle Cell Disease – Pipeline by Pfizer 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
      • Syros Pharmaceuticals Inc
        • Table Sickle Cell Disease – Pipeline by Syros Pharmaceuticals Inc
  • Dormant Projects
    • Thalassemia – Dormant Projects
      • Table Thalassemia – Dormant Projects
    • Hemophilia B – Dormant Projects
      • Table Hemophilia B – Dormant Projects
    • Sickle Cell Disease – Dormant Projects
      • Table Sickle Cell Disease – Dormant Projects
  • Discontinued Products
    • Thalassemia – Discontinued Products
      • Table Thalassemia – Discontinued Products
    • Hemophilia B – Discontinued Products
      • Table Hemophilia B – Discontinued Products
  • Product Development Milestones
    • Thalassemia – Product Development Milestones
      • Featured News & Press Releases
    • Hemophilia B – Product Development Milestones
      • Featured News & Press Releases
    • Sickle Cell Disease – Product Development Milestones
      • Featured News & Press Releases
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

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