Table of Contents Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Ornithine-Transcarbamylase Deficiency Overview 6 Therapeutics Development 7 Pipeline Products for Ornithine-Transcarbamylase Deficiency - Overview 7 Ornithine-Transcarbamylase Deficiency - Therapeutics under Development by Companies 8 Ornithine-Transcarbamylase Deficiency - Pipeline Products Glance 9 Late Stage Products 9 Early Stage Products 10 Ornithine-Transcarbamylase Deficiency - Products under Development by Companies 11 Ornithine-Transcarbamylase Deficiency - Companies Involved in Therapeutics Development 12 Dimension Therapeutics Inc 12 Lucane Pharma SA 13 PhaseRx Inc 14 Promethera Biosciences SA 15 Unicyte AG 16 Ornithine-Transcarbamylase Deficiency - Therapeutics Assessment 17 Assessment by Monotherapy Products 17 Assessment by Target 18 Assessment by Mechanism of Action 19 Assessment by Route of Administration 21 Assessment by Molecule Type 22 Drug Profiles 24 Cell Therapy for Urea Cycle Disorders and Liver Failure - Drug Profile 24 Product Description 24 Mechanism Of Action 24 R&D Progress 24 DTX-301 - Drug Profile 26 Product Description 26 Mechanism Of Action 26 R&D Progress 26 HepaStem - Drug Profile 28 Product Description 28 Mechanism Of Action 28 R&D Progress 28 PRX-OTC - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 sodium benzoate - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 Stem Cell Therapy for Type 1 Diabetes, Urea Cycle Disorders and Acute Liver Failure - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Ornithine-Transcarbamylase Deficiency - Dormant Projects 34 Ornithine-Transcarbamylase Deficiency - Product Development Milestones 35 Featured News & Press Releases 35 Nov 28, 2016: PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency 35 Appendix 36 Methodology 36 Coverage 36 Secondary Research 36 Primary Research 36 Expert Panel Validation 36 Contact Us 36 Disclaimer 37

List of Tables Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2016 7 Number of Products under Development by Companies, H2 2016 8 Comparative Analysis by Late Stage Development, H2 2016 9 Comparative Analysis by Early Stage Development, H2 2016 10 Products under Development by Companies, H2 2016 11 Ornithine-Transcarbamylase Deficiency - Pipeline by Dimension Therapeutics Inc, H2 2016 12 Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, H2 2016 13 Ornithine-Transcarbamylase Deficiency - Pipeline by PhaseRx Inc, H2 2016 14 Ornithine-Transcarbamylase Deficiency - Pipeline by Promethera Biosciences SA, H2 2016 15 Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, H2 2016 16 Assessment by Monotherapy Products, H2 2016 17 Number of Products by Stage and Target, H2 2016 18 Number of Products by Stage and Mechanism of Action, H2 2016 20 Number of Products by Stage and Route of Administration, H2 2016 21 Number of Products by Stage and Molecule Type, H2 2016 23 Ornithine-Transcarbamylase Deficiency - Dormant Projects, H2 2016 34 List of Figures Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2016 7 Number of Products under Development by Companies, H2 2016 8 Comparative Analysis by Late Stage Development, H2 2016 9 Comparative Analysis by Early Stage Products, H2 2016 10 Assessment by Monotherapy Products, H2 2016 17 Number of Products by Stage and Targets, H2 2016 18 Number of Products by Mechanism of Actions, H2 2016 19 Number of Products by Stage and Mechanism of Actions, H2 2016 19 Number of Products by Stage and Routes of Administration, H2 2016 21 Number of Products by Molecule Types, H2 2016 22 Number of Products by Stage and Molecule Types, H2 2016 22

Dimension Therapeutics Inc Lucane Pharma SA PhaseRx Inc Promethera Biosciences SA Unicyte AG