Many serious diseases, including primary sclerosing cholangitis and chronic kidney disease, are caused by the dysfunction of membrane proteins, which perform critical functions such as substrate transport and signal transduction across membranes. Current treatments do not directly address the underlying cause of these diseases, which is the reduced activity or inability of these proteins to reach the cell membrane.

Rectify Pharmaceuticals is developing Positive Functional Modulators (PFMs), a novel class of oral, small molecule therapies designed to rectify membrane protein dysfunction. Their proprietary PFM product platform enables the discovery of first- and best-in-class small molecule therapies that restore and enhance membrane protein function. The platform has demonstrated success in drugging multiple ATP-binding cassette (ABC) transporters and has broad applicability to other classes of membrane proteins. Rectify's lead candidate, RTY-694, is a dual-acting PFM that improves bile composition and enhances bile flow, addressing both cholestasis and cholangitis associated with multiple hepatobiliary diseases.

Orally available small molecule therapeutics

Targets ATP-binding cassette (ABC) transporters and other membrane proteins

Lead candidate RTY-694 is a dual-acting Positive Functional Modulator

RTY-694 improves bile composition and enhances bile flow

Proprietary PFM product platform enables efficient and rapid drug discovery

Platform applicable to a wide range of diseases caused by protein dysfunction

The primary target audience includes patients suffering from diseases caused by membrane protein dysfunction, such as primary sclerosing cholangitis (PSC), chronic kidney disease (CKD), and other cardio-renal-metabolic diseases.