Table of Contents Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Fanconi Anemia Overview 6 Therapeutics Development 7 Pipeline Products for Fanconi Anemia - Overview 7 Pipeline Products for Fanconi Anemia - Comparative Analysis 8 Fanconi Anemia - Therapeutics under Development by Companies 9 Fanconi Anemia - Therapeutics under Investigation by Universities/Institutes 10 Fanconi Anemia - Pipeline Products Glance 11 Clinical Stage Products 11 Early Stage Products 12 Fanconi Anemia - Products under Development by Companies 13 Fanconi Anemia - Products under Investigation by Universities/Institutes 14 Fanconi Anemia - Companies Involved in Therapeutics Development 15 Abeona Therapeutics, Inc. 15 Genethon 16 Fanconi Anemia - Therapeutics Assessment 17 Assessment by Monotherapy Products 17 Assessment by Target 18 Assessment by Mechanism of Action 20 Assessment by Route of Administration 22 Assessment by Molecule Type 24 Drug Profiles 26 ABO-301 - Drug Profile 26 Product Description 26 Mechanism of Action 26 R&D Progress 26 AD-6626 - Drug Profile 27 Product Description 27 Mechanism of Action 27 R&D Progress 27 Cell Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile 28 Product Description 28 Mechanism of Action 28 R&D Progress 28 JP4-039 - Drug Profile 29 Product Description 29 Mechanism of Action 29 R&D Progress 29 Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 33 Product Description 33 Mechanism of Action 33 R&D Progress 33 Fanconi Anemia - Recent Pipeline Updates 34 Fanconi Anemia - Dormant Projects 35 Fanconi Anemia - Product Development Milestones 36 Featured News & Press Releases 36 Feb 24, 2016: Abeona Therapeutics to Present on CRISPR/CAS9 Technology Platform Progress at CRISPR Precision Gene Editing Congress 36 Oct 03, 2011: Experimental Drug Could Help Protect Some Cancer Patients From Radiation Side Effects, UPCI Researchers Find 36 Appendix 38 Methodology 38 Coverage 38 Secondary Research 38 Primary Research 38 Expert Panel Validation 38 Contact Us 38 Disclaimer 39

List of Tables Number of Products under Development for Fanconi Anemia, H1 2016 7 Number of Products under Development for Fanconi Anemia - Comparative Analysis, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Number of Products under Investigation by Universities/Institutes, H1 2016 10 Comparative Analysis by Clinical Stage Development, H1 2016 11 Comparative Analysis by Early Stage Development, H1 2016 12 Products under Development by Companies, H1 2016 13 Products under Investigation by Universities/Institutes, H1 2016 14 Fanconi Anemia - Pipeline by Abeona Therapeutics, Inc. , H1 2016 15 Fanconi Anemia - Pipeline by Genethon, H1 2016 16 Assessment by Monotherapy Products, H1 2016 17 Number of Products by Stage and Target, H1 2016 19 Number of Products by Stage and Mechanism of Action, H1 2016 21 Number of Products by Stage and Route of Administration, H1 2016 23 Number of Products by Stage and Molecule Type, H1 2016 25 Fanconi Anemia Therapeutics - Recent Pipeline Updates, H1 2016 34 Fanconi Anemia - Dormant Projects, H1 2016 35 List of Figures Number of Products under Development for Fanconi Anemia, H1 2016 7 Number of Products under Development for Fanconi Anemia - Comparative Analysis, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Number of Products under Investigation by Universities/Institutes, H1 2016 10 Comparative Analysis by Clinical Stage Development, H1 2016 11 Comparative Analysis by Early Stage Products, H1 2016 12 Assessment by Monotherapy Products, H1 2016 17 Number of Products by Targets, H1 2016 18 Number of Products by Stage and Targets, H1 2016 18 Number of Products by Mechanism of Actions, H1 2016 20 Number of Products by Stage and Mechanism of Actions, H1 2016 20 Number of Products by Routes of Administration, H1 2016 22 Number of Products by Stage and Routes of Administration, H1 2016 22 Number of Products by Molecule Types, H1 2016 24 Number of Products by Stage and Molecule Types, H1 2016 24

Abeona Therapeutics, Inc. Genethon