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

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 631.2 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

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