Charlotte Barker, Editor, BioInsights, speaks to Annarita Miccio, Group Leader, Imagine Institute of Genetic Diseases, discussing ongoing research aimed at developing gene therapies for β-hemoglobinopathies, focusing on transcriptional and epigenetic regulation. They also explore innovative tools such as CRISPR-Cas9, base editing, and epigenome editing to improve treatment safety and efficiency, with the goal of making gene therapies more accessible globally.