Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

  • ID: 4663535
  • Report
  • 143 Pages
  • GBI Research
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FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Bristol-Myers Squibb Co
  • Epizyme Inc
  • Ionis Pharmaceuticals Inc
  • NKT Therapeutics Inc
  • RegenxBio Inc
  • MORE

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
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Bristol-Myers Squibb Co
  • Epizyme Inc
  • Ionis Pharmaceuticals Inc
  • NKT Therapeutics Inc
  • RegenxBio Inc
  • MORE

1 Table of Contents
1.1 List of Tables
1.2 List of Figures

2 Introduction
2.1 Hereditary Hematological Disorders Report Coverage
2.2 Thalassemia - Overview
2.3 Hemophilia B - Overview
2.4 Sickle Cell Disease - Overview

3 Therapeutics Development
3.1 Thalassemia
3.2 Hemophilia B - Therapeutics Development
3.3 Sickle Cell Disease - Therapeutics Development

4 Therapeutics Assessment
4.1 Thalassemia - Therapeutics Assessment
4.2 Hemophilia B - Therapeutics Assessment
4.3 Sickle Cell Disease - Therapeutics Assessment

5 Companies Involved in Therapeutics Development
5.1 Thalassemia - Companies Involved in Therapeutics Development
5.2 Hemophilia B - Companies Involved in Therapeutics Development
5.3 Sickle Cell Disease - Companies Involved in Therapeutics Development

6 Dormant Projects
6.1 Thalassemia - Dormant Projects
6.2 Hemophilia B - Dormant Projects
6.3 Sickle Cell Disease - Dormant Projects

7 Discontinued Products
7.1 Thalassemia - Discontinued Products
7.2 Hemophilia B - Discontinued Products

8 Product Development Milestones
8.1 Thalassemia - Product Development Milestones
8.2 Hemophilia B - Product Development Milestones
8.3 Sickle Cell Disease - Product Development Milestones

9 Appendix
9.1 Methodology
9.2 Coverage
9.3 Secondary Research
9.4 Primary Research
9.5 Expert Panel Validation
9.6 Contact Us
9.7 Disclaimer

List of Tables
Table 1: Number of Products under Development for Thalassemia
Table 2: Number of Products under Development by Companies, Thalassemia
Table 3: Number of Products under Development by Universities/Institutes, Thalassemia
Table 4: Products under Development by Companies, Thalassemia
Table 5: Products under Development by Universities/Institutes, Thalassemia
Table 6: Number of Products under Development for Hemophilia B
Table 7: Number of Products under Development by Companies, Hemophilia B
Table 8: Number of Products under Development by Universities/Institutes, Hemophilia B
Table 9: Products under Development by Companies, Hemophilia B
Table 10: Products under Development by Universities/Institutes, Hemophilia B
Table 11: Number of Products under Development for Sickle Cell Disease
Table 12: Number of Products under Development by Companies, Sickle Cell Disease
Table 13: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Table 14: Products under Development by Companies, Sickle Cell Disease
Table 15: Products under Development by Universities/Institutes, Sickle Cell Disease
Table 16: Number of Products by Stage and Target, Thalassemia
Table 17: Number of Products by Stage and Mechanism of Action, Thalassemia
Table 18: Number of Products by Stage and Route of Administration, Thalassemia
Table 19: Number of Products by Stage and Molecule Type, Thalassemia
Table 20: Number of Products by Stage and Target, Hemophilia B
Table 21: Number of Products by Stage and Mechanism of Action, Hemophilia B
Table 22: Number of Products by Stage and Route of Administration, Hemophilia B
Table 23: Number of Products by Stage and Molecule Type, Hemophilia B
Table 24: Number of Products by Stage and Target, H2 2018, Sickle Cell Disease
Table 25: Number of Products by Stage and Mechanism of Action, Sickle Cell Disease
Table 26: Number of Products by Stage and Route of Administration, Sickle Cell Disease
Table 27: Number of Products by Stage and Molecule Type, Sickle Cell Disease
Table 28: Thalassemia - Pipeline by Acceleron Pharma Inc
Table 29: Thalassemia - Pipeline by Agios Pharmaceuticals Inc
Table 30: Thalassemia - Pipeline by bluebird bio Inc
Table 31: Thalassemia - Pipeline by Cadila Healthcare Ltd,
Table 32: Thalassemia - Pipeline by Calimmune Inc
Table 33: Thalassemia - Pipeline by CRISPR Therapeutics
Table 34: Thalassemia - Pipeline by Editas Medicine Inc
Table 35: Thalassemia - Pipeline by Errant Gene Therapeutics LLC
Table 36: Thalassemia - Pipeline by Gamida Cell Ltd
Table 37: Thalassemia - Pipeline by Gilead Sciences Inc
Table 38: Thalassemia - Pipeline by Incyte Corp
Table 39: Thalassemia - Pipeline by Ionis Pharmaceuticals Inc
Table 40: Thalassemia - Pipeline by Kiadis Pharma NV
Table 41: Thalassemia - Pipeline by La Jolla Pharmaceutical Company
Table 42: Thalassemia - Pipeline by Merck & Co Inc
Table 43: Thalassemia - Pipeline by Poseida Therapeutics Inc
Table 44: Thalassemia - Pipeline by Protagonist Therapeutics Inc
Table 45: Thalassemia - Pipeline by Sangamo Therapeutics Inc
Table 46: Thalassemia - Pipeline by Silence Therapeutics Plc
Table 47: Thalassemia - Pipeline by Vifor Pharma AG
Table 48: Hemophilia B - Pipeline by Amarna Therapeutics BV
Table 49: Hemophilia B - Pipeline by Bayer AG
Table 50: Hemophilia B - Pipeline by Bioverativ Inc
Table 51: Hemophilia B - Pipeline by Catalyst Biosciences Inc
Table 52: Hemophilia B - Pipeline by China Biologic Products Inc
Table 53: Hemophilia B - Pipeline by CSL Ltd

List of Figures
Figure 1: Number of Products under Development for Thalassemia
Figure 2: Number of Products under Development by Companies, Thalassemia
Figure 3: Number of Products under Development by Universities/Institutes, Thalassemia
Figure 4: Number of Products under Development for Hemophilia B
Figure 5: Number of Products under Development by Companies, Hemophilia B
Figure 6: Number of Products under Development by Universities/Institutes, Hemophilia B
Figure 7: Number of Products under Development for Sickle Cell Disease
Figure 8: Number of Products under Development by Companies, Sickle Cell Disease
Figure 9: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Figure 10: Number of Products by Top 10 Targets, Thalassemia
Figure 11: Number of Products by Stage and Top 10 Targets, Thalassemia
Figure 12: Number of Products by Top 10 Mechanism of Actions, Thalassemia
Figure 13: Number of Products by Stage and Top 10 Mechanism of Actions, Thalassemia
Figure 14: Number of Products by Routes of Administration, Thalassemia
Figure 15: Number of Products by Stage and Routes of Administration, Thalassemia
Figure 16: Number of Products by Molecule Types, Thalassemia
Figure 17: Number of Products by Stage and Molecule Types, Thalassemia
Figure 18: Number of Products by Targets, Hemophilia B
Figure 19: Number of Products by Stage and Targets, Hemophilia B
Figure 20: Number of Products by Mechanism of Actions, Hemophilia B
Figure 21: Number of Products by Stage and Mechanism of Actions, Hemophilia B
Figure 22: Number of Products by Routes of Administration, Hemophilia B
Figure 23: Number of Products by Stage and Routes of Administration, Hemophilia B
Figure 24: Number of Products by Molecule Types, Hemophilia B
Figure 25: Number of Products by Stage and Molecule Types, Hemophilia B
Figure 26: Number of Products by Top 10 Targets, Sickle Cell Disease
Figure 27: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease
Figure 28: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 29: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 30: Number of Products by Routes of Administration, Sickle Cell Disease
Figure 31: Number of Products by Stage and Routes of Administration, Sickle Cell Disease
Figure 32: Number of Products by Top 10 Molecule Types, Sickle Cell Disease
Figure 33: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease 48

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  • Acceleron Pharma Inc
  • Agios Pharmaceuticals Inc
  • Amarna Therapeutics BV
  • Angiocrine Bioscience Inc
  • Annexin Pharmaceuticals AB
  • ArQule Inc
  • Bayer AG
  • Bio Products Laboratory Ltd
  • Bioverativ Inc
  • Bluebird bio Inc
  • Bristol-Myers Squibb Co
  • CRISPR Therapeutics
  • CSL Ltd
  • Cadila Healthcare Ltd
  • Calimmune Inc
  • Catalyst Biosciences Inc
  • Cell Source Inc
  • China Biologic Products Inc
  • Editas Medicine Inc
  • Epizyme Inc
  • Errant Gene Therapeutics LLC
  • Expression Therapeutics LLC
  • Fulcrum Therapeutics Inc
  • GC Pharma
  • Gamida Cell Ltd
  • Gilead Sciences Inc
  • Global Blood Therapeutics Inc
  • Homology Medicines, Inc.
  • Incyte Corp
  • Intellia Therapeutics Inc
  • Ionis Pharmaceuticals Inc
  • Ironwood Pharmaceuticals Inc
  • Kiadis Pharma NV
  • LFB SA
  • La Jolla Pharmaceutical Company
  • Logicbio Therapeutics Inc
  • MaxCyte Inc
  • Merck & Co Inc
  • MimeTech Srl
  • Morphogenesis Inc
  • NKT Therapeutics Inc
  • Novartis AG
  • Novo Nordisk AS
  • OPKO Biologics Ltd
  • Orphagen Pharmaceuticals Inc
  • Oryzon Genomics SA
  • Pharming Group NV
  • Poseida Therapeutics Inc
  • Prolong Pharmaceuticals LLC
  • Promethera Biosciences SA
  • Protagonist Therapeutics Inc
  • RegenxBio Inc
  • ReveraGen BioPharma Inc
  • Sancilio & Company Inc
  • Sangamo Therapeutics Inc
  • Sanofi
  • Shire Plc
  • Silence Therapeutics Plc
  • Spark Therapeutics Inc
  • Syros Pharmaceuticals Inc
  • UniQure NV
  • Vifor Pharma AG
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