MC4 receptor pharmacochaperone

Domain Therapeutics (rebranded as Kainova Therapeutics in 2026) is developing a small molecule pharmacochaperone targeting the melanocortin-4 (MC4) receptor for the treatment of rare metabolic diseases and obesity. The MC4 receptor is a G protein-coupled receptor (GPCR) that plays a central role in energy homeostasis and appetite regulation. Genetic mutations in the MC4R gene often result in misfolded proteins that are retained within the endoplasmic reticulum, leading to receptor deficiency at the cell surface. This pharmacochaperone is designed to bind and stabilize these misfolded receptors, facilitating their proper folding and trafficking to the plasma membrane to restore functional signaling. The program is being developed in collaboration with the Université de Montréal and Merck KGaA.

Other names

MC4 pharmacochaperoneMC-4 pharmacochaperoneMC 4 pharmacochaperone