sodium phenylbutyrate + genistein

PTI G-4660 is an investigational combination therapy consisting of sodium phenylbutyrate and genistein, primarily studied for the treatment of cystic fibrosis in patients homozygous for the Delta F508 mutation. Sodium phenylbutyrate acts as a chemical chaperone and histone deacetylase (HDAC) inhibitor that promotes the trafficking of the misfolded Delta F508-CFTR protein from the endoplasmic reticulum to the cell surface. Genistein, a naturally occurring isoflavone, serves as a CFTR potentiator, enhancing the chloride channel activity of the CFTR protein once it reaches the plasma membrane. This dual-mechanism approach was designed to address both the trafficking defect and the gating defect associated with the most common cystic fibrosis mutation. The combination was evaluated in clinical trials led by the University of Pennsylvania and the Children's Hospital of Philadelphia.

Other names

Phenylbutyrate/Genistein DuotherapyPhenylbutyrate and Genistein