The Cystic Fibrosis Foundation has awarded Icagen Inc. up to $11 million for a multi-year drug discovery program to identify and develop treatments for people with cystic fibrosis who have nonsense mutations.
Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly or not made at all. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. This leads to a shorter, incomplete protein that either does not work or is unstable and is quickly destroyed by the cell.
With the funding from the CF Foundation, Icagen plans to screen more than 2 million compounds to determine if they can enable the protein-making machinery of the cell to override these premature stop signals so that a full-length, functional protein can be made.
Icagen also will use computer modeling to simulate how millions of drug candidates might work to suppress the nonsense mutations in the CFTR gene. The company plans to use the computer modeling process -- a method intended to speed up drug discovery -- to identify and develop “families of molecules” that are suitable to be developed into drugs.
About 5 percent of people with CF carry two mutations, such as nonsense mutations, that do not generate a full-length CFTR protein. Because of this, they are unlikely to benefit solely from existing CFTR modulator therapies, medications that specifically target the dysfunctional protein.