Gene Therapy for Hemophilia: Innovations, Milestones, and Future Prospects

Haemophilia, a genetic bleeding disorder predominantly affecting males, arises from deficiencies or dysfunctions in clotting factors VIII or IX. Conventional treatments, such as frequent intravenous infusions of clotting factor concentrates, pose challenges in terms of cost, accessibility, and patient adherence. Gene therapy offers a transformative solution by correcting genetic mutations, enabling the body to produce clotting factors endogenously. Indian scientists have recently pioneered advances in this field, employing adeno-associated viral vectors for efficient and safe gene delivery. This approach addresses limitations of traditional therapies, including the development of inhibitors and the need for repetitive infusions. With the integration of cutting-edge genome editing technologies, such as CRISPR/Cas9, hemophilia treatment is poised for long-term efficacy and improved quality of life. Despite these promising developments, challenges remain, including affordability, ethical considerations, and equitable access. This paper highlights the latest breakthroughs, clinical advancements, and future directions in gene therapy for hemophilia, emphasizing India's significant contributions to global research. The findings underscore the potential of gene therapy to shift hemophilia management from routine care to curative strategies, redefining therapeutic possibilities for this chronic condition.