Global Hereditary Hematological Disorders Drug Development Pipeline Review 2018

1/9/2019, 4:35 PM (Source: GlobeNewswire)

Dublin, Jan. 09, 2019 (GLOBE NEWSWIRE) -- The "Hereditary Hematological Disorders Drug Development Pipeline Review, 2018" report has been added to's offering.

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.

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.


  • 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?

Key Topics Covered:

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

Companies Mentioned

  • 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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Related Topics: Drug Discovery, Hematological Drugs 


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