Sickle Cell Disease Drug Development Pipeline Review, 2017

Pages: 77
Published: November 2017
Report Code: GBIHC025IDB
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This report provides an overview of the Sickle Cell Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for sickle cell anemia and vaso-occlusive crisis associated with sickle cell disease, and features dormant and discontinued projects.

Sickle cell anemia is a genetic (inherited) 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. There are a total of 62 products in development for this indication, by 45 companies and nine academic institutions. Hemoglobin and a range of histone deacetylases are the most common molecular targets being studied in sickle cell anemia.

Vaso-occlusive crisis associated with sickle cell disease is a painful episode that occurs in people who have sickle cell anemia. Symptoms include fever, pain, fatigue, abdominal pain and swelling, headaches, shortness of breath, dehydration and infection. There are a total of 10 products in development for this indication.

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

1 Table of Contents

1 Table of Contents 4

1.1 List of Tables 5

1.2 List of Figures 6

2 Introduction 7

2.1 Sickle Cell Disease Report Coverage 7

2.2 Sickle Cell Disease – Overview 7

2.3 Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Overview 7

3 Therapeutics Development 8

3.1 Sickle Cell Disease 8

3.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 15

4 Therapeutics Assessment 18

4.1 Sickle Cell Disease 18

4.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 25

5 Companies Involved in Therapeutics Development 31

5.1 Sickle Cell Disease 31

5.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 47

6 Dormant Projects 52

6.1 Sickle Cell Disease 52

6.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 54

7 Discontinued Products 55

7.1 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 55

8 Product Development Milestones 56

8.1 Sickle Cell Disease 56

8.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease 67

9 Appendix 76

9.1 Methodology 76

9.2 Coverage 76

9.3 Secondary Research 76

9.4 Primary Research 76

9.5 Expert Panel Validation 76

9.6 Contact Us 77

9.7 Disclaimer 77

List of Tables

1.1 List of Tables

Table 1: Number of Products under Development for Sickle Cell Disease 8

Table 2: Number of Products under Development by Companies, Sickle Cell Disease 9

Table 3: Number of Products under Development by Universities/Institutes, Sickle Cell Disease 11

Table 4: Products under Development by Companies, Sickle Cell Disease 12

Table 5: Products under Development by Universities/Institutes, Sickle Cell Disease 14

Table 6: Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease 15

Table 7: Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 16

Table 8: Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 17

Table 9: Number of Products by Stage and Target, Sickle Cell Disease 19

Table 10: Number of Products by Stage and Mechanism of Action, Sickle Cell Disease 21

Table 11: Number of Products by Stage and Route of Administration, Sickle Cell Disease 23

Table 12: Number of Products by Stage and Molecule Type, Sickle Cell Disease 24

Table 13: Number of Products by Stage and Target, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 26

Table 14: Number of Products by Stage and Mechanism of Action, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 27

Table 15: Number of Products by Stage and Route of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 29

Table 16: Number of Products by Stage and Molecule Type, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 30

Table 17: Sickle Cell Disease – Pipeline by Acceleron Pharma Inc 31

Table 18: Sickle Cell Disease – Pipeline by Addmedica SAS 31

Table 19: Sickle Cell Disease – Pipeline by Angiocrine Bioscience Inc 32

Table 20: Sickle Cell Disease – Pipeline by Annexin Pharmaceuticals AB 32

Table 21: Sickle Cell Disease – Pipeline by ArQule Inc 33

Table 22: Sickle Cell Disease – Pipeline by Bio Products Laboratory Ltd 33

Table 23: Sickle Cell Disease – Pipeline by Bioverativ Inc 34

Table 24: Sickle Cell Disease – Pipeline by bluebird bio Inc 35

Table 25: Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Co 35

Table 26: Sickle Cell Disease – Pipeline by Calimmune Inc 36

Table 27: Sickle Cell Disease – Pipeline by CRISPR Therapeutics 36

Table 28: Sickle Cell Disease – Pipeline by CSL Ltd 37

Table 29: Sickle Cell Disease – Pipeline by Daiichi Sankyo Co Ltd 37

Table 30: Sickle Cell Disease – Pipeline by Editas Medicine Inc 38

Table 31: Sickle Cell Disease – Pipeline by Errant Gene Therapeutics LLC 38

Table 32: Sickle Cell Disease – Pipeline by Gamida Cell Ltd 38

Table 33: Sickle Cell Disease – Pipeline by Genethon SA 39

Table 34: Sickle Cell Disease – Pipeline by Gilead Sciences Inc 39

Table 35: Sickle Cell Disease – Pipeline by Global Blood Therapeutics Inc 40

Table 36: Sickle Cell Disease – Pipeline by Incyte Corp 40

Table 37: Sickle Cell Disease – Pipeline by Ironwood Pharmaceuticals Inc 41

Table 38: Sickle Cell Disease – Pipeline by La Jolla Pharmaceutical Company 41

Table 39: Sickle Cell Disease – Pipeline by MaxCyte Inc 42

Table 40: Sickle Cell Disease – Pipeline by Merck & Co Inc 43

Table 41: Sickle Cell Disease – Pipeline by Morphogenesis Inc 43

Table 42: Sickle Cell Disease – Pipeline by NKT Therapeutics Inc 43

Table 43: Sickle Cell Disease – Pipeline by Novartis AG 44

Table 44: Sickle Cell Disease – Pipeline by Orphagen Pharmaceuticals Inc 44

Table 45: Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals LLC 45

Table 46: Sickle Cell Disease – Pipeline by Protagonist Therapeutics Inc 45

Table 47: Sickle Cell Disease – Pipeline by ReveraGen BioPharma Inc 46

Table 48: Sickle Cell Disease – Pipeline by Sancilio & Company Inc 46

Table 49: Sickle Cell Disease – Pipeline by Sangamo Therapeutics Inc 46

Table 50: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by AstraZeneca Plc 47

Table 51: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Co 48

Table 52: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Gilead Sciences Inc 48

Table 53: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Modus Therapeutics Holding AB 49

Table 54: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Novartis AG 49

Table 55: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by NuvOx Pharma LLC 50

Table 56: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Pfizer Inc 50

Table 57: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals LLC 51

Table 58: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Seattle Genetics Inc 51

Table 59: Sickle Cell Disease – Dormant Projects 52

Table 60: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Dormant Projects 54

Table 61: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Discontinued Products 55

List of Figures

1.2 List of Figures

Figure 1: Number of Products under Development for Sickle Cell Disease 8

Figure 2: Number of Products under Development by Companies, Sickle Cell Disease 9

Figure 3: Number of Products under Development by Universities/Institutes, Sickle Cell Disease 11

Figure 4: Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease 15

Figure 5: Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 16

Figure 6: Number of Products by Top 10 Targets, Sickle Cell Disease 18

Figure 7: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease 18

Figure 8: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease 20

Figure 9: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease 20

Figure 10: Number of Products by Routes of Administration, Sickle Cell Disease 22

Figure 11: Number of Products by Stage and Routes of Administration, Sickle Cell Disease 22

Figure 12: Number of Products by Top 10 Molecule Types, Sickle Cell Disease 23

Figure 13: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease 24

Figure 14: Number of Products by Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 25

Figure 15: Number of Products by Stage and Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 25

Figure 16: Number of Products by Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 26

Figure 17: Number of Products by Stage and Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 27

Figure 18: Number of Products by Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 28

Figure 19: Number of Products by Stage and Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 28

Figure 20: Number of Products by Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 29

Figure 21: Number of Products by Stage and Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease 30

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