Addressing Gaps In Cystic Fibrosis Treatment By Fixing Nonsense Mutations

By Alexandra Crawley, PH.D., Co-founder and Principal Investigator, Life Edit Therapeutics, Inc.

Cell and gene therapies are enabling treatment for a number of severe or life-threatening diseases with previously unmet medical needs. Recently approved therapies for cystic fibrosis (CF) – a life-limiting disease caused by the inheritance of two recessive mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – are able to stabilize a mutant protein, allowing it to function more normally.

However, about 7% of CF cases involve nonsense mutations in the CFTR gene, and current therapies cannot normalize the protein’s function, leaving CF patients who have these mutations with no therapeutic options. Examine how access to the world’s largest and most diverse collection of novel RGNs and deaminases is increasing the potential for addressing the needs of CF patients with nonsense mutations by helping their cells produce functional CFTR protein.