Rectify Pharmaceuticals develops Positive Functional Modulators (PFMs), small molecules designed to rectify membrane protein dysfunction that contributes to serious diseases such as primary sclerosing cholangitis and chronic kidney disease. Their lead candidate, RTY-694, targets bile composition and flow to mitigate the effects of cholestasis and cholangitis in hepatobiliary diseases.
Funding
$100M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.



Founders
Product
Problem
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.
Solution
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.
Target Audience
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.
Features
- 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