1 Table of Contents 1 Table of Contents 4 1.1 List of Tables 5 1.2 List of Figures 6 2 Introduction 8 2.1 Mucopolysaccharidosis Disorders Report Coverage 8 2.2 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 8 2.3 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Overview 8 2.4 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview 8 3 Therapeutics Development 9 3.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9 3.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14 3.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19 4 Therapeutics Assessment 23 4.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23 4.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28 4.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34 5 Companies Involved in Therapeutics Development 40 5.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 40 5.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 44 5.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 49 6 Dormant Projects 54 6.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 54 6.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 55 6.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 56 7 Discontinued Products 57 7.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 57 7.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 57 7.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 57 8 Product Development Milestones 58 8.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 58 8.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 70 8.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 82 9 Appendix 97 9.1 Methodology 97 9.2 Coverage 97 9.3 Secondary Research 97 9.4 Primary Research 97 9.5 Expert Panel Validation 97 9.6 Contact Us 98 9.7 Disclaimer 98

1.1 List of Tables Table 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9 Table 2: Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 10 Table 3: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 11 Table 4: Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 12 Table 5: Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 13 Table 6: Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14 Table 7: Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 15 Table 8: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 16 Table 9: Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 17 Table 10: Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 18 Table 11: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19 Table 12: Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 20 Table 13: Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 21 Table 14: Number of Products by Stage and Target Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23 Table 15: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 25 Table 16: Number of Products by Stage and Route of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 26 Table 17: Number of Products by Stage and Molecule Type Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 28 Table 18: Number of Products by Stage and Target Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 29 Table 19: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 30 Table 20: Number of Products by Stage and Route of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 32 Table 21: Number of Products by Stage and Molecule Type Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 33 Table 22: Number of Products by Stage and Target Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 35 Table 23: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 36 Table 24: Number of Products by Stage and Route of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 38 Table 25: Number of Products by Stage and Molecule Type Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 39 Table 26: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc 40 Table 27: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Inc 40 Table 28: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by CRISPR Therapeutics 41 Table 29: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Eloxx Pharmaceuticals Inc 41 Table 30: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Immusoft Corp 42 Table 31: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd 42 Table 32: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health Inc 43 Table 33: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by RegenxBio Inc 43 Table 34: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Sangamo Therapeutics Inc 44 Table 35: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by AngioChem Inc 44 Table 36: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by ArmaGen Inc 45 Table 37: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Denali Therapeutics Inc 45 Table 38: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by GC Pharma 46 Table 39: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd 47 Table 40: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA 47 Table 41: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by RegenxBio Inc 48 Table 42: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Sangamo Therapeutics Inc 48 Table 43: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Shire Plc 49 Table 44: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Abeona Therapeutics Inc 50 Table 45: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by ArmaGen Inc 50 Table 46: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Axcentua Pharmaceuticals AB 51 Table 47: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by BioMarin Pharmaceutical Inc 51 Table 48: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA 52 Table 49: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Lysogene SAS 52 Table 50: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Swedish Orphan Biovitrum AB 53 Table 51: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects 54 Table 52: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Dormant Projects 55 Table 53: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects 56 Table 54: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Discontinued Products 57 Table 55: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Discontinued Products 57 Table 56: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Discontinued Products 571.2 List of Figures Figure 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9 Figure 2: Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 10 Figure 3: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 11 Figure 4: Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14 Figure 5: Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 15 Figure 6: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 16 Figure 7: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19 Figure 8: Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 20 Figure 9: Number of Products by Stage and Targets Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23 Figure 10: Number of Products by Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 24 Figure 11: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 24 Figure 12: Number of Products by Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 25 Figure 13: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 26 Figure 14: Number of Products by Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 27 Figure 15: Number of Products by Stage and Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 27 Figure 16: Number of Products by Stage and Targets Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28 Figure 17: Number of Products by Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 29 Figure 18: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 30 Figure 19: Number of Products by Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 31 Figure 20: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 31 Figure 21: Number of Products by Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 32 Figure 22: Number of Products by Stage and Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 33 Figure 23: Number of Products by Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34 Figure 24: Number of Products by Stage and Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34 Figure 25: Number of Products by Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 35 Figure 26: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 36 Figure 27: Number of Products by Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 37 Figure 28: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 37 Figure 29: Number of Products by Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 38 Figure 30: Number of Products by Stage and Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 39

Abeona Therapeutics Inc AngioChem Inc ArmaGen Inc Axcentua Pharmaceuticals AB BioMarin Pharmaceutical Inc CRISPR Therapeutics Denali Therapeutics Inc Eloxx Pharmaceuticals Inc GC Pharma Immusoft Corp JCR Pharmaceuticals Co Ltd Laboratorios Del Dr Esteve SA Lysogene SAS Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) OPKO Health Inc RegenxBio Inc Sangamo Therapeutics Inc Shire Plc Swedish Orphan Biovitrum AB