Table of Contents Table of Contents 2 List of Tables 6 List of Figures 7 Introduction 8 Global Markets Direct Report Coverage 8 Alpha- Antitrypsin Deficiency Overview 9 Therapeutics Development 10 Pipeline Products for Alpha- Antitrypsin Deficiency - Overview 10 Alpha- Antitrypsin Deficiency - Therapeutics under Development by Companies 11 Alpha- Antitrypsin Deficiency - Pipeline Products Glance 13 Late Stage Products 13 Clinical Stage Products 14 Early Stage Products 15 Alpha- Antitrypsin Deficiency - Products under Development by Companies 16 Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 18 Alnylam Pharmaceuticals, Inc. 18 Annapurna Therapeutics 19 Applied Genetic Technologies Corporation 20 Arrowhead Research Corporation 21 Carolus Therapeutics, Inc. 22 Cevec Pharmaceuticals GmbH 23 Dicerna Pharmaceuticals, Inc. 24 Digna Biotech, S.L. 25 Editas Medicine, Inc. 26 Grifols, S.A. 27 iBio, Inc. 28 Inhibrx 29 International Stem Cell Corporation 30 Ionis Pharmaceuticals, Inc. 31 Kamada Ltd. 32 Polyphor Ltd. 33 ProMetic Life Sciences Inc. 34 rEVO Biologics, Inc. 35 Sangamo BioSciences, Inc. 36 Alpha- Antitrypsin Deficiency - Therapeutics Assessment 37 Assessment by Monotherapy Products 37 Assessment by Target 38 Assessment by Mechanism of Action 40 Assessment by Route of Administration 42 Assessment by Molecule Type 44 Drug Profiles 46 ALN-AAT - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 alpha-1 proteinase inhibitor (human) - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 alpha-1 proteinase inhibitor (human) second generation - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 ANN-001 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 ARC-AAT - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 CT-2009 - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 DB-027 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 Drugs for Alpha-1 Antitrypsin Deficiency - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 ISIS-AATRx - Drug Profile 59 Product Description 59 Mechanism of Action 59 R&D Progress 59 POL-6014 - Drug Profile 60 Product Description 60 Mechanism of Action 60 R&D Progress 60 rAAV2-CB-hAAT - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 Recombinant A1PI - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 Recombinant Alpha-1 Antitrypsin Replacement for Alpha-Antitrypsin Deficiency - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 Recombinant Human Alpha-1 Antitrypsin - Drug Profile 66 Product Description 66 Mechanism of Action 66 R&D Progress 66 Recombinant Human Alpha-1 Antitrypsin - Drug Profile 67 Product Description 67 Mechanism of Action 67 R&D Progress 67 Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile 68 Product Description 68 Mechanism of Action 68 R&D Progress 68 RNAi Oligonucleotide for Alpha- Antitrypsin Deficiency and Liver Disease - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 71 Product Description 71 Mechanism of Action 71 R&D Progress 71 Alpha- Antitrypsin Deficiency - Recent Pipeline Updates 72 Alpha- Antitrypsin Deficiency - Dormant Projects 80 Alpha- Antitrypsin Deficiency - Discontinued Products 81 Alpha- Antitrypsin Deficiency - Product Development Milestones 82 Featured News & Press Releases 82 Feb 25, 2016: Dicerna Reports Progress with GalNAc-DsiRNA-EX-Conjugates 82 Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency 83 Jan 19, 2016: Arrowhead's ARC-AAT Granted EMA Orphan Drug Designation 83 Dec 08, 2015: Kamada Completes Enrollment of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of AAT Deficiency 84 Nov 09, 2015: Sangamo BioSciences Announces Publication Of Improved Method For Efficient Targeted Integration In Hematopoietic Stem Cells And T-cells 84 Jul 08, 2015: Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand 85 Jul 02, 2015: Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency 85 Jun 10, 2015: Arrowhead Receives Orphan Drug Designation for ARC-AAT 86 May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency 87 May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT 88 Appendix 90 Methodology 90 Coverage 90 Secondary Research 90 Primary Research 90 Expert Panel Validation 90 Contact Us 90 Disclaimer 91

List of Tables Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2016 10 Number of Products under Development by Companies, H1 2016 12 Comparative Analysis by Late Stage Development, H1 2016 13 Comparative Analysis by Clinical Stage Development, H1 2016 14 Comparative Analysis by Early Stage Development, H1 2016 15 Products under Development by Companies, H1 2016 16 Products under Development by Companies, H1 2016 (Contd..1) 17 Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 18 Alpha- Antitrypsin Deficiency - Pipeline by Annapurna Therapeutics, H1 2016 19 Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H1 2016 20 Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Research Corporation, H1 2016 21 Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H1 2016 22 Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H1 2016 23 Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H1 2016 24 Alpha- Antitrypsin Deficiency - Pipeline by Digna Biotech, S.L., H1 2016 25 Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine, Inc., H1 2016 26 Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H1 2016 27 Alpha- Antitrypsin Deficiency - Pipeline by iBio, Inc., H1 2016 28 Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H1 2016 29 Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H1 2016 30 Alpha- Antitrypsin Deficiency - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 31 Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H1 2016 32 Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H1 2016 33 Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc., H1 2016 34 Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, Inc., H1 2016 35 Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H1 2016 36 Assessment by Monotherapy Products, H1 2016 37 Number of Products by Stage and Target, H1 2016 39 Number of Products by Stage and Mechanism of Action, H1 2016 41 Number of Products by Stage and Route of Administration, H1 2016 43 Number of Products by Stage and Molecule Type, H1 2016 45 Alpha- Antitrypsin Deficiency Therapeutics - Recent Pipeline Updates, H1 2016 72 Alpha- Antitrypsin Deficiency - Dormant Projects, H1 2016 80 Alpha- Antitrypsin Deficiency - Discontinued Products, H1 2016 81 List of Figures Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2016 10 Number of Products under Development by Companies, H1 2016 11 Comparative Analysis by Clinical Stage Development, H1 2016 14 Comparative Analysis by Early Stage Products, H1 2016 15 Assessment by Monotherapy Products, H1 2016 37 Number of Products by Targets, H1 2016 38 Number of Products by Stage and Targets, H1 2016 38 Number of Products by Mechanism of Actions, H1 2016 40 Number of Products by Stage and Mechanism of Actions, H1 2016 40 Number of Products by Routes of Administration, H1 2016 42 Number of Products by Stage and Routes of Administration, H1 2016 42 Number of Products by Molecule Types, H1 2016 44 Number of Products by Stage and Molecule Types, H1 2016 44

Alnylam Pharmaceuticals, Inc. Annapurna Therapeutics Applied Genetic Technologies Corporation Arrowhead Research Corporation Carolus Therapeutics, Inc. Cevec Pharmaceuticals GmbH Dicerna Pharmaceuticals, Inc. Digna Biotech, S.L. Editas Medicine, Inc. Grifols, S.A. iBio, Inc. Inhibrx International Stem Cell Corporation Ionis Pharmaceuticals, Inc. Kamada Ltd. Polyphor Ltd. ProMetic Life Sciences Inc. rEVO Biologics, Inc. Sangamo BioSciences, Inc.