Hemophilia gene therapy-New country initiatives

摘要: Gene therapy is an opportunity for haemophilia patients to receive a one-time treatment and have lasting factor levels years or decades instead of dependence on repeated administration within short intervals sustained supply drug. Great strides been made in the development gene last decade. Adeno-associated virus (AAV) vector-mediated transfer A B has entered phase III trial stage. by lentiviral vector editing technologies using VIII (FVIII) IX (FIX) genes are now entering clinical evaluation. It expected that first FVIII FIX products will soon be approved distributed major markets. Global access critical goal. This review presents new ongoing efforts towards this goal countries other than North America Europe. In Japan, researchers, regulators funders established promising platform multiple diseases including haemophilia. Decades scientific research China led recently registered AAV-mediated B. Other earlier phases building programmes participate international trials. 2 feasibility low- middle-income aims demonstrate could become available resource-constrained socio-economic settings. The different strategies establishing provide opportunities closing global gap care.