Orphi Therapeutics Inc

Orphi Therapeutics is an early-stage biotechnology company based in Carlsbad, California, developing small-molecule pharmacological chaperone therapies for rare monogenic diseases, including MC4R-linked monogenic obesity and the GM2 gangliosidoses (Tay-Sachs and Sandhoff disease). Pharmacological chaperones selectively bind and stabilize target proteins to improve folding, reduce degradation, and restore enzyme function, offering a novel therapeutic approach for these underserved rare-disease patient populations. The company has received NIH SBIR/STTR Phase I and Phase II funding to advance its lead chaperone programs.