Gene therapy for cystic fibrosis in the clinic

Country

United Kingdom

A new gene therapy for cystic fibrosis has entered clinical development in the UK with the aim of improving outcomes for people with cystic fibrosis regardless of their gene mutation. The trial specifically focuses on adults with cystic fibrosis who cannot benefit from the current modulators of the membrane protein – cystic fibrosis transmembrane conductance regulator (CFTR).