First in class disease-modifying agents for Cystic Fibrosis
FoldRx has developed a high throughput assay to detect small molecules that rescue a yeast mutant defective in endoplasmic reticulum (ER) to Golgi trafficking. These activators of protein trafficking have shown to be potent functional correctors of the mistrafficked protein DF508-CFTR, the main cause of cystic fibrosis. Further characterization and optimization around these correctors to identify disease-modifying agents for the treatment of CF have been initiated at FoldRx in partnership with the Cystic Fibrosis Foundation. FoldRx expects to identify its first clinical corrector candidate for the treatment of CF in 2009.
For more information on the Cystic Fibrosis Foundation go to http://www.cff.org/.