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 Fanconi Anemia - Therapeutics Development 7 Pipeline Overview 7 Pipeline by Companies 8 Pipeline by Universities/Institutes 9 Products under Development by Companies 10 Products under Development by Universities/Institutes 11 Fanconi Anemia - Therapeutics Assessment 12 Assessment by Target 12 Assessment by Mechanism of Action 14 Assessment by Route of Administration 16 Assessment by Molecule Type 18 Fanconi Anemia - Companies Involved in Therapeutics Development 20 Abeona Therapeutics Inc 20 Genethon SA 20 Novartis AG 20 Fanconi Anemia - Drug Profiles 22 ABO-301 - Drug Profile 22 Product Description 22 Mechanism Of Action 22 R&D Progress 22 AD-6626 - Drug Profile 23 Product Description 23 Mechanism Of Action 23 R&D Progress 23 eltrombopag olamine - Drug Profile 24 Product Description 24 Mechanism Of Action 24 R&D Progress 24 EXG-34217 - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 Fancalen - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 JP4-039 - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Small Molecules for Fanconi Anemia - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 Stem Cell Therapy for Fanconi Anemia - Drug Profile 35 Product Description 35 Mechanism Of Action 35 R&D Progress 35 Stem Cell Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 Fanconi Anemia - Dormant Projects 39 Fanconi Anemia - Product Development Milestones 40 Featured News & Press Releases 40 Feb 24, 2016: Abeona Therapeutics to Present on CRISPR/CAS9 Technology Platform Progress at CRISPR Precision Gene Editing Congress 40 Oct 03, 2011: Experimental Drug Could Help Protect Some Cancer Patients From Radiation Side Effects, UPCI Researchers Find 40 Appendix 42 Methodology 42 Coverage 42 Secondary Research 42 Primary Research 42 Expert Panel Validation 42 Contact Us 42 Disclaimer 43

List of Tables Number of Products under Development for Fanconi Anemia, H2 2017 7 Number of Products under Development by Companies, H2 2017 8 Number of Products under Development by Universities/Institutes, H2 2017 9 Products under Development by Companies, H2 2017 10 Products under Development by Universities/Institutes, H2 2017 11 Number of Products by Stage and Target, H2 2017 13 Number of Products by Stage and Mechanism of Action, H2 2017 15 Number of Products by Stage and Route of Administration, H2 2017 17 Number of Products by Stage and Molecule Type, H2 2017 19 Fanconi Anemia - Pipeline by Abeona Therapeutics Inc, H2 2017 20 Fanconi Anemia - Pipeline by Genethon SA, H2 2017 20 Fanconi Anemia - Pipeline by Novartis AG, H2 2017 21 Fanconi Anemia - Dormant Projects, H2 2017 39 List of Figures Number of Products under Development for Fanconi Anemia, H2 2017 7 Number of Products under Development by Companies, H2 2017 8 Number of Products under Development by Universities/Institutes, H2 2017 9 Number of Products by Targets, H2 2017 12 Number of Products by Stage and Targets, H2 2017 12 Number of Products by Mechanism of Actions, H2 2017 14 Number of Products by Stage and Mechanism of Actions, H2 2017 14 Number of Products by Routes of Administration, H2 2017 16 Number of Products by Stage and Routes of Administration, H2 2017 16 Number of Products by Molecule Types, H2 2017 18 Number of Products by Stage and Molecule Types, H2 2017 18

Abeona Therapeutics Inc Genethon SA Novartis AG