Currently focused on developing novel therapies for MC4R-linked monogenic obesity and Sandhoff/Tay Sachs disease, an early-stage biotech structured around development of pharmacological chaperones (PCs) for treatment of monogenic diseases, Orphi Therapeutics Inc specializes in early-stage biotechnology research and therapeutic advancements, PCs are the small molecules modulating protein homeostasis of a specific protein target: they increase cellular levels of the protein target by increasing the folding and trafficking efficiency with a concomitant reduction in premature degradation. OrPhi is
