Table of Contents Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Gaucher Disease Overview 8 Therapeutics Development 9 Pipeline Products for Gaucher Disease - Overview 9 Pipeline Products for Gaucher Disease - Comparative Analysis 10 Gaucher Disease - Therapeutics under Development by Companies 11 Gaucher Disease - Therapeutics under Investigation by Universities/Institutes 12 Gaucher Disease - Pipeline Products Glance 13 Clinical Stage Products 13 Early Stage Products 14 Gaucher Disease - Products under Development by Companies 15 Gaucher Disease - Products under Investigation by Universities/Institutes 16 Gaucher Disease - Companies Involved in Therapeutics Development 17 Actelion Ltd 17 Genzyme Corp 18 greenovation Biotech GmbH 19 JCR Pharmaceuticals Co Ltd 20 Lixte Biotechnology Holdings Inc 21 Lysosomal Therapeutics Inc 22 Neuraltus Pharmaceuticals Inc 23 Pharming Group NV 24 Protalix BioTherapeutics Inc 25 Sangamo BioSciences Inc 26 Shire Plc 27 The International Biotechnology Center (IBC) Generium 28 Gaucher Disease - Therapeutics Assessment 29 Assessment by Monotherapy Products 29 Assessment by Target 30 Assessment by Mechanism of Action 32 Assessment by Route of Administration 34 Assessment by Molecule Type 36 Drug Profiles 38 ADN-LYS - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 ambroxol - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 Gene Therapy for Gaucher Disease - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 ibiglustat - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 imiglucerase biosimilar - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 LB-201 - Drug Profile 44 Product Description 44 Mechanism Of Action 44 R&D Progress 44 LB-205 - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 miglustat - Drug Profile 46 Product Description 46 Mechanism Of Action 46 R&D Progress 46 MOSS-GBA - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 NCGC-607 - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 NP-003 - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 PRX-112 - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 SBLSD-3 - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile 60 Product Description 60 Mechanism Of Action 60 R&D Progress 60 taliglucerase alfa - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 velaglucerase alfa - Drug Profile 62 Product Description 62 Mechanism Of Action 62 R&D Progress 62 Gaucher Disease - Dormant Projects 63 Gaucher Disease - Discontinued Products 65 Gaucher Disease - Product Development Milestones 66 Featured News & Press Releases 66 Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 66 Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 66 Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 67 Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 67 Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 68 Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 69 Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 70 Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 70 Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease 71 Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72 Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72 Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV 73 Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease 74 Nov 22, 2011: Shire Announces Regulatory Filings In US And EU For New Manufacturing Facility 74 Mar 21, 2011: Shire Presents Data For VPRIV In Gaucher Disease At 2011 ACMG Annual Meeting 75 Appendix 76 Methodology 76 Coverage 76 Secondary Research 76 Primary Research 76 Expert Panel Validation 76 Contact Us 76 Disclaimer 77

List of Tables Number of Products under Development for Gaucher Disease, H2 2016 9 Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10 Number of Products under Development by Companies, H2 2016 11 Number of Products under Investigation by Universities/Institutes, H2 2016 12 Comparative Analysis by Clinical Stage Development, H2 2016 13 Comparative Analysis by Early Stage Development, H2 2016 14 Products under Development by Companies, H2 2016 15 Products under Investigation by Universities/Institutes, H2 2016 16 Gaucher Disease - Pipeline by Actelion Ltd, H2 2016 17 Gaucher Disease - Pipeline by Genzyme Corp, H2 2016 18 Gaucher Disease - Pipeline by greenovation Biotech GmbH, H2 2016 19 Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 20 Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H2 2016 21 Gaucher Disease - Pipeline by Lysosomal Therapeutics Inc, H2 2016 22 Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H2 2016 23 Gaucher Disease - Pipeline by Pharming Group NV, H2 2016 24 Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H2 2016 25 Gaucher Disease - Pipeline by Sangamo BioSciences Inc, H2 2016 26 Gaucher Disease - Pipeline by Shire Plc, H2 2016 27 Gaucher Disease - Pipeline by The International Biotechnology Center (IBC) Generium, H2 2016 28 Assessment by Monotherapy Products, H2 2016 29 Number of Products by Stage and Target, H2 2016 31 Number of Products by Stage and Mechanism of Action, H2 2016 33 Number of Products by Stage and Route of Administration, H2 2016 35 Number of Products by Stage and Molecule Type, H2 2016 37 Gaucher Disease - Dormant Projects, H2 2016 63 Gaucher Disease - Dormant Projects (Contd..1), H2 2016 64 Gaucher Disease - Discontinued Products, H2 2016 65 List of Figures Number of Products under Development for Gaucher Disease, H2 2016 9 Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10 Number of Products under Development by Companies, H2 2016 11 Number of Products under Investigation by Universities/Institutes, H2 2016 12 Comparative Analysis by Clinical Stage Development, H2 2016 13 Comparative Analysis by Early Stage Products, H2 2016 14 Assessment by Monotherapy Products, H2 2016 29 Number of Products by Targets, H2 2016 30 Number of Products by Stage and Targets, H2 2016 30 Number of Products by Top 10 Mechanism of Actions, H2 2016 32 Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 32 Number of Products by Routes of Administration, H2 2016 34 Number of Products by Stage and Routes of Administration, H2 2016 34 Number of Products by Molecule Types, H2 2016 36 Number of Products by Stage and Molecule Types, H2 2016 36

Actelion Ltd Genzyme Corp greenovation Biotech GmbH JCR Pharmaceuticals Co Ltd Lixte Biotechnology Holdings Inc Lysosomal Therapeutics Inc Neuraltus Pharmaceuticals Inc Pharming Group NV Protalix BioTherapeutics Inc Sangamo BioSciences Inc Shire Plc The International Biotechnology Center (IBC) Generium