Introduction Executive Summary Beta-thalassaemia: Overview • Causes • Mechanism of Action • Signs and Symptoms • Diagnosis • Disease Management Pipeline Therapeutics • Comparative Analysis Therapeutic Assessment • Assessment by Product Type • Assessment by Stage and Product Type • Assessment by Route of Administration • Assessment by Stage and Route of Administration • Assessment by Molecule Type • Assessment by Stage and Molecule Type Beta-thalassaemia – DelveInsight’s Analytical Perspective Mid Stage Products (Phase II) • Comparative Analysis IMR-687: Imara, Inc. • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Early Stage Products (Phase I) • Comparative Analysis BD 211: Shanghai BDgene • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Preclinical and Discovery Stage Products • Comparative Analysis DISC a: Disc Medicine • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Inactive Products • Comparative Analysis Beta-thalassaemia Key Companies Beta-thalassaemia Key Products Beta-thalassaemia- Unmet Needs Beta-thalassaemia- Market Drivers and Barriers Beta-thalassaemia- Future Perspectives and Conclusion Beta-thalassaemia Analyst Views Beta-thalassaemia Key Companies Appendix

Table 1 Total Products for Beta-thalassaemia Table 2 Late Stage Products Table 3 Mid Stage Products Table 4 Early Stage Products Table 5 Pre-clinical & Discovery Stage Products Table 6 Assessment by Product Type Table 7 Assessment by Stage and Product Type Table 8 Assessment by Route of Administration Table 9 Assessment by Stage and Route of Administration Table 10 Assessment by Molecule Type Table 11 Assessment by Stage and Molecule Type Table 12 Inactive Products

• CRISPR Therapeutics • Imara, Inc. • Sangamo Therapeutics • Vifor Pharma • Bioray Laboratories • EdiGene (GuangZhou) Inc. • Ionis Pharmaceuticals, Inc. • Agios Pharmaceuticals, Inc. • Silence Therapeutics plc • Aruvant Sciences • Phoenicia Biosciences • San Rocco Therapeutics • Shanghai BDgene • Beam Therapeutics • EmeraMed • Regenacy Pharmaceuticals • Editas Medicine • Fulcrum Therapeutics • Allife Medical Science and Technology • Global Blood Therapeutics, Inc • Orchard Therapeutics • Acceleron Pharma • Disc Medicine