This program aimed to identify potential therapies for people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Overriding these premature stop signals would allow full-length, functional CFTR protein to be made.

Status

No further development in CF is planned at this time.

Sponsor

This program was sponsored by Icagen Inc. and partially funded by the Cystic Fibrosis Foundation.