Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

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.

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.

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

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

Bristol-Myers Squibb Co

Cadila Healthcare Ltd

Calimmune Inc

Catalyst Biosciences Inc

Cell Source Inc

China Biologic Products Inc

Epizyme Inc

Expression Therapeutics LLC

Fulcrum Therapeutics Inc

GC Pharma

Global Blood Therapeutics Inc

Homology Medicines, Inc.

Intellia Therapeutics Inc

Ionis Pharmaceuticals Inc

Ironwood Pharmaceuticals Inc

Kiadis Pharma NV

LFB SA

Logicbio Therapeutics Inc

MaxCyte 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

RegenxBio Inc

ReveraGen BioPharma Inc

Sancilio & Company Inc

Sanofi

Shire Plc

Silence Therapeutics Plc

Spark Therapeutics Inc

Syros Pharmaceuticals Inc

UniQure NV

Vifor Pharma AG

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

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

7 Discontinued Products 98

7.1 Thalassemia - Discontinued Products 98

7.2 Hemophilia B - Discontinued Products 98

8 Product Development Milestones 99

8.1 Thalassemia - Product Development Milestones 99

8.2 Hemophilia B - Product Development Milestones 114

8.3 Sickle Cell Disease - Product Development Milestones 127

9 Appendix 142

9.1 Methodology 142

9.2 Coverage 142

9.3 Secondary Research 142

9.4 Primary Research 142

9.5 Expert Panel Validation 142

9.6 Contact Us 143

9.7 Disclaimer 143

List of Tables

1.1 List of Tables

Table 1: Number of Products under Development for Thalassemia 9

Table 2: Number of Products under Development by Companies, Thalassemia 10

Table 3: Number of Products under Development by Universities/Institutes, Thalassemia 12

Table 4: Products under Development by Companies, Thalassemia 12

Table 5: Products under Development by Universities/Institutes, Thalassemia 14

Table 6: Number of Products under Development for Hemophilia B 15

Table 7: Number of Products under Development by Companies, Hemophilia B 16

Table 8: Number of Products under Development by Universities/Institutes, Hemophilia B 17

Table 9: Products under Development by Companies, Hemophilia B 18

Table 10: Products under Development by Universities/Institutes, Hemophilia B 20

Table 11: Number of Products under Development for Sickle Cell Disease 21

Table 12: Number of Products under Development by Companies, Sickle Cell Disease 22

Table 13: Number of Products under Development by Universities/Institutes, Sickle Cell Disease 24

Table 14: Products under Development by Companies, Sickle Cell Disease 24

Table 15: Products under Development by Universities/Institutes, Sickle Cell Disease 27

Table 16: Number of Products by Stage and Target, Thalassemia 30

Table 17: Number of Products by Stage and Mechanism of Action, Thalassemia 32

Table 18: Number of Products by Stage and Route of Administration, Thalassemia 34

Table 19: Number of Products by Stage and Molecule Type, Thalassemia 35

Table 20: Number of Products by Stage and Target, Hemophilia B 37

Table 21: Number of Products by Stage and Mechanism of Action, Hemophilia B 38

Table 22: Number of Products by Stage and Route of Administration, Hemophilia B 40

Table 23: Number of Products by Stage and Molecule Type, Hemophilia B 41

Table 24: Number of Products by Stage and Target, H2 2018, Sickle Cell Disease 43

Table 25: Number of Products by Stage and Mechanism of Action, Sickle Cell Disease 45

Table 26: Number of Products by Stage and Route of Administration, Sickle Cell Disease 47

Table 27: Number of Products by Stage and Molecule Type, Sickle Cell Disease 48

Table 28: Thalassemia – Pipeline by Acceleron Pharma Inc 49

Table 29: Thalassemia – Pipeline by Agios Pharmaceuticals Inc 50

Table 30: Thalassemia – Pipeline by bluebird bio Inc 50

Table 31: Thalassemia – Pipeline by Cadila Healthcare Ltd, 51

Table 32: Thalassemia – Pipeline by Calimmune Inc 51

Table 33: Thalassemia – Pipeline by CRISPR Therapeutics 52

Table 34: Thalassemia – Pipeline by Editas Medicine Inc 53

Table 35: Thalassemia – Pipeline by Errant Gene Therapeutics LLC 53

Table 36: Thalassemia – Pipeline by Gamida Cell Ltd 53

Table 37: Thalassemia – Pipeline by Gilead Sciences Inc 54

Table 38: Thalassemia – Pipeline by Incyte Corp 55

Table 39: Thalassemia – Pipeline by Ionis Pharmaceuticals Inc 55

Table 40: Thalassemia – Pipeline by Kiadis Pharma NV 56

Table 41: Thalassemia – Pipeline by La Jolla Pharmaceutical Company 56

Table 42: Thalassemia – Pipeline by Merck & Co Inc 57

Table 43: Thalassemia – Pipeline by Poseida Therapeutics Inc 57

Table 44: Thalassemia – Pipeline by Protagonist Therapeutics Inc 58

Table 45: Thalassemia – Pipeline by Sangamo Therapeutics Inc 58

Table 46: Thalassemia – Pipeline by Silence Therapeutics Plc 59

Table 47: Thalassemia – Pipeline by Vifor Pharma AG 59

Table 48: Hemophilia B – Pipeline by Amarna Therapeutics BV 60

Table 49: Hemophilia B – Pipeline by Bayer AG 60

Table 50: Hemophilia B – Pipeline by Bioverativ Inc 61

Table 51: Hemophilia B – Pipeline by Catalyst Biosciences Inc 62

Table 52: Hemophilia B – Pipeline by China Biologic Products Inc 62

Table 53: Hemophilia B – Pipeline by CSL Ltd 63

List of Figures

1.2 List of Figures

Figure 1: Number of Products under Development for Thalassemia 9

Figure 2: Number of Products under Development by Companies, Thalassemia 10

Figure 3: Number of Products under Development by Universities/Institutes, Thalassemia 11

Figure 4: Number of Products under Development for Hemophilia B 14

Figure 5: Number of Products under Development by Companies, Hemophilia B 15

Figure 6: Number of Products under Development by Universities/Institutes, Hemophilia B 17

Figure 7: Number of Products under Development for Sickle Cell Disease 20

Figure 8: Number of Products under Development by Companies, Sickle Cell Disease 21

Figure 9: Number of Products under Development by Universities/Institutes, Sickle Cell Disease 23

Figure 10: Number of Products by Top 10 Targets, Thalassemia 28

Figure 11: Number of Products by Stage and Top 10 Targets, Thalassemia 29

Figure 12: Number of Products by Top 10 Mechanism of Actions, Thalassemia 31

Figure 13: Number of Products by Stage and Top 10 Mechanism of Actions, Thalassemia 31

Figure 14: Number of Products by Routes of Administration, Thalassemia 33

Figure 15: Number of Products by Stage and Routes of Administration, Thalassemia 33

Figure 16: Number of Products by Molecule Types, Thalassemia 34

Figure 17: Number of Products by Stage and Molecule Types, Thalassemia 35

Figure 18: Number of Products by Targets, Hemophilia B 36

Figure 19: Number of Products by Stage and Targets, Hemophilia B 36

Figure 20: Number of Products by Mechanism of Actions, Hemophilia B 37

Figure 21: Number of Products by Stage and Mechanism of Actions, Hemophilia B 38

Figure 22: Number of Products by Routes of Administration, Hemophilia B 39

Figure 23: Number of Products by Stage and Routes of Administration, Hemophilia B 39

Figure 24: Number of Products by Molecule Types, Hemophilia B 40

Figure 25: Number of Products by Stage and Molecule Types, Hemophilia B 41

Figure 26: Number of Products by Top 10 Targets, Sickle Cell Disease 42

Figure 27: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease 42

Figure 28: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease 44

Figure 29: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease 44

Figure 30: Number of Products by Routes of Administration, Sickle Cell Disease 46

Figure 31: Number of Products by Stage and Routes of Administration, Sickle Cell Disease 46

Figure 32: Number of Products by Top 10 Molecule Types, Sickle Cell Disease 47

Figure 33: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease 48

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