New Research on Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

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This report 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.

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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?

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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

Table of Contents

1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 5
1.2 List of Figures 7

2 Introduction 8
2.1 Hereditary Hematological Disorders Report Coverage 8
2.2 Thalassemia – Overview 8
2.3 Hemophilia B – Overview 8
2.4 Sickle Cell Disease – Overview 8

3 Therapeutics Development 9
3.1 Thalassemia 9
3.2 Hemophilia B – Therapeutics Development 14
3.3 Sickle Cell Disease – Therapeutics Development 20

4 Therapeutics Assessment 28
4.1 Thalassemia – Therapeutics Assessment 28
4.2 Hemophilia B – Therapeutics Assessment 36
4.3 Sickle Cell Disease – Therapeutics Assessment 42

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5 Companies Involved in Therapeutics Development 49
5.1 Thalassemia – Companies Involved in Therapeutics Development 49
5.2 Hemophilia B – Companies Involved in Therapeutics Development 59
5.3 Sickle Cell Disease – Companies Involved in Therapeutics Development 72

6 Dormant Projects 93
6.1 Thalassemia – Dormant Projects 93
6.2 Hemophilia B – Dormant Projects 94
6.3 Sickle Cell Disease – Dormant Projects 95

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