5^th International Conference on Rare Diseases and Orphan Drugs

Gene Therapy and Regenerative Medicine: Gene therapy aims to treat or prevent disease by modifying genetic material or introducing genetic instructions into cells, while regenerative medicine seeks to repair, replace, or restore damaged cells, tissues, or biological functions. These approaches are particularly relevant to selected rare diseases in which a defined genetic defect or loss of cellular function contributes to the condition. This session examines gene delivery systems, genome editing, cell-based therapies, tissue engineering, safety assessment, clinical translation, and the scientific challenges involved in developing advanced treatments.