New Technologies Empower Hemophilic Arthropathy Treatment: Focusing on Regenerative Medicine, Molecular Targeting, and Gene Therapy

ABSTRACT Hemophilic arthropathy (HA), a major complication of hemophilia, is a chronic osteoarthropathy driven by repeated joint bleeding. Although traditional therapies such as coagulation factor replacement, physical rehabilitation, and surgery can manage symptoms, they cannot fundamentally repair established joint damage or break the pathological cascades. This review systematically summarizes therapeutic frontiers in HA, including regenerative medicine, molecular targeting, and gene therapy. Regenerative medicine aims to restore joint function through tissue repair using mesenchymal stem cells and their exosomes, as well as multifunctional biomaterials such as nanocarriers and hydrogels for targeted delivery and microenvironment modulation. Molecular targeting offers precision treatment by interfering with key pathological pathways using monoclonal antibodies that either improve coagulation or target pro‐inflammatory factors. Non‐coding RNAs are also being explored for their roles in regulating inflammation and cartilage degradation. Gene therapy, a paradigm shift from symptomatic care to fundamental repair, enables continuous endogenous production of coagulation factors, thereby preventing bleeding and delaying HA progression. The successful translation of these therapies into clinical practice requires overcoming challenges such as optimizing cell survival in hostile environments, standardizing biomaterial production, managing immune responses to vectors, and reducing costs. Future research should focus on interdisciplinary strategies, personalized treatment plans, and long‐term follow‐up studies to achieve synergistic effects and ensure safety. These innovative approaches promise to significantly improve joint function and quality of life for HA patients, ultimately working toward a functional cure.