Table of Contents Table of Contents 2 List of Tables 5 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) Overview 7 Therapeutics Development 8 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Stage of Development 8 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Therapy Area 9 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Indication 10 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Products Glance 11 Late Stage Products 11 Early Stage Products 12 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Companies 13 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Therapeutics Assessment 15 Assessment by Monotherapy/Combination Products 15 Assessment by Mechanism of Action 16 Assessment by Route of Administration 18 Assessment by Molecule Type 20 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Companies Involved in Therapeutics Development 22 AngioChem Inc. 22 ArmaGen Inc. 23 Bioasis Technologies Inc. 24 Green Cross Corporation 25 JCR Pharmaceuticals Co., Ltd. 26 Laboratorios Del Dr. Esteve S.A. 27 RegenxBio Inc. 28 Sangamo BioSciences, Inc. 29 Shire Plc 30 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Drug Profiles 31 AGT-182 - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 EGT-301 - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 idursulfase - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 idursulfase beta - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 JR-032 - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 JR-141 - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 MTf-I2S - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 MTfp-I2S - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 RGX-121 - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 SB-913 - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Featured News & Press Releases 47 Jul 14, 2016: ArmaGen Presents Data from First Cohort of Phase 1/2a of AGT-182 for Treatment of Hunter Syndrome 47 Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121 48 Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II 48 Apr 19, 2016: Preclinical Data from REGENXBIO’s RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting 49 Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome 50 Jul 31, 2015: biOasis Announces the Successful Delivery to the CNS of an Enzyme used to Treat Hunter Syndrome 51 Jun 23, 2015: ACTSI-supports first-in-man clinical trial for Hunter syndrome at Emory University Hospital 51 Jun 15, 2015: ArmaGen Achieves Clinical Milestone in Hunter Syndrome Collaboration with Shire 52 Mar 10, 2015: PacificGMP and ArmaGen Announce Successful Manufacture of ArmaGen's investigational enzyme replacement therapy, AGT-182, for Phase 1 Clinical Trial 53 Jan 26, 2015: Shire's Investigational SHP609, idursulfase-IT, Receives FDA Fast Track Designation for the Treatment of Neurocognitive Decline Associated with Hunter Syndrome 54 Dec 11, 2014: ArmaGen Announces FDA Acceptance of IND Application for AGT-182 for the Treatment of Hunter Syndrome 54 Sep 18, 2014: biOasis Initiates MPS II Study with Renowned Lysosomal Storage Disease Expert Dr. Maurizio Scarpa and The Brains for Brain Foundation 55 Jun 17, 2014: ArmaGen Receives Notice of Allowance for U.S. Patent Covering Novel Investigational Therapy for Hunter Syndrome 56 Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria 56 Jul 18, 2013: ARMAGEN Receives US Orphan Designation For Lead Product AGT-182 57 Appendix 58 Methodology 58 Coverage 58 Secondary Research 58 Primary Research 58 Expert Panel Validation 58 Contact Us 58 Disclaimer 59

Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Review, H2 2016 Summary Global Markets Direct’s, ‘Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Review, H2 2016’, provides in depth analysis on Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted pipeline therapeutics. The report provides comprehensive information on the Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) , targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics development and features dormant and discontinued projects. Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data. Scope - The report provides a snapshot of the global therapeutic landscape for Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - The report reviews Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources - The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages - The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics and enlists all their major and minor projects - The report assesses Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type - The report summarizes all the dormant and discontinued pipeline projects - The report reviews latest news and deals related to Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics Reasons to buy - Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Identify and understand the targeted therapy areas and indications for Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Identify the use of drugs for target identification and drug repurposing - Identify potential new clients or partners in the target demographic - Develop strategic initiatives by understanding the focus areas of leading companies - Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics - Devise corrective measures for pipeline projects by understanding Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) development landscape - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scopeTable of Contents Table of Contents 2 List of Tables 5 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) Overview 7 Therapeutics Development 8 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Stage of Development 8 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Therapy Area 9 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Indication 10 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Products Glance 11 Late Stage Products 11 Early Stage Products 12 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Products under Development by Companies 13 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Therapeutics Assessment 15 Assessment by Monotherapy/Combination Products 15 Assessment by Mechanism of Action 16 Assessment by Route of Administration 18 Assessment by Molecule Type 20 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Companies Involved in Therapeutics Development 22 AngioChem Inc. 22 ArmaGen Inc. 23 Bioasis Technologies Inc. 24 Green Cross Corporation 25 JCR Pharmaceuticals Co., Ltd. 26 Laboratorios Del Dr. Esteve S.A. 27 RegenxBio Inc. 28 Sangamo BioSciences, Inc. 29 Shire Plc 30 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Drug Profiles 31 AGT-182 - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 EGT-301 - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 idursulfase - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 idursulfase beta - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 JR-032 - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 JR-141 - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 MTf-I2S - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 MTfp-I2S - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 RGX-121 - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 SB-913 - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Featured News & Press Releases 47 Jul 14, 2016: ArmaGen Presents Data from First Cohort of Phase 1/2a of AGT-182 for Treatment of Hunter Syndrome 47 Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121 48 Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II 48 Apr 19, 2016: Preclinical Data from REGENXBIO’s RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting 49 Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome 50 Jul 31, 2015: biOasis Announces the Successful Delivery to the CNS of an Enzyme used to Treat Hunter Syndrome 51 Jun 23, 2015: ACTSI-supports first-in-man clinical trial for Hunter syndrome at Emory University Hospital 51 Jun 15, 2015: ArmaGen Achieves Clinical Milestone in Hunter Syndrome Collaboration with Shire 52 Mar 10, 2015: PacificGMP and ArmaGen Announce Successful Manufacture of ArmaGen's investigational enzyme replacement therapy, AGT-182, for Phase 1 Clinical Trial 53 Jan 26, 2015: Shire's Investigational SHP609, idursulfase-IT, Receives FDA Fast Track Designation for the Treatment of Neurocognitive Decline Associated with Hunter Syndrome 54 Dec 11, 2014: ArmaGen Announces FDA Acceptance of IND Application for AGT-182 for the Treatment of Hunter Syndrome 54 Sep 18, 2014: biOasis Initiates MPS II Study with Renowned Lysosomal Storage Disease Expert Dr. Maurizio Scarpa and The Brains for Brain Foundation 55 Jun 17, 2014: ArmaGen Receives Notice of Allowance for U.S. Patent Covering Novel Investigational Therapy for Hunter Syndrome 56 Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria 56 Jul 18, 2013: ARMAGEN Receives US Orphan Designation For Lead Product AGT-182 57 Appendix 58 Methodology 58 Coverage 58 Secondary Research 58 Primary Research 58 Expert Panel Validation 58 Contact Us 58 Disclaimer 59

AngioChem Inc. ArmaGen Inc. Bioasis Technologies Inc. Green Cross Corporation JCR Pharmaceuticals Co., Ltd. Laboratorios Del Dr. Esteve S.A. RegenxBio Inc. Sangamo BioSciences, Inc. Shire Plc