Gene Editing for Haemophilia—The Next Frontier

via HDR in B lymphocytes, and this promising ex-vivo gene therapy entered a Phase 1/2 trial. Base and prime editors have also been successfully exploited in cellular models to precisely correct gene defects but their translational potential is limited by the many diverse haemophilia-causing genetic variants that need to be addressed. As these technologies mature, rigorous long-term follow-up and safety monitoring will be essential to offer patients a definitive cure for haemophilia.