Rectifying function, Restoring hope
See how we are applying our PFM product platform to rectify protein dysfunction that drives primary sclerosing cholangitis (PSC), cardio-renal-metabolic diseases, and other serious diseases.
About us
We develop Positive Functional Modulators (PFMs), a novel class of disease-modifying oral, small molecules specifically designed to rectify the dysfunction of membrane proteins. Our PFMs directly target the underlying cause of serious diseases mediated by membrane protein dysfunction.
Our PFM product platform
Membrane proteins perform several critical functions, including mediating transport of substrates and signal transduction across membranes.
Dysfunctional Membrane Proteins
Dysfunction in these proteins––resulting from reduced activity or inability to reach the cell membrane––can lead to a wide array of debilitating and serious diseases across multiple therapeutic areas. Rectifying protein dysfunction with small molecules could ameliorate many diseases, but this has historically been challenging.
Functional Membrane Proteins
Rectify built a proprietary PFM product platform that enables efficient and rapid discovery of first- and best-in-class small molecule therapies that rectify membrane protein dysfunction. Our industry-leading platform drives the discovery of PFMs to restore and enhance membrane protein function.
The platform demonstrated success in drugging multiple ATP-binding cassette (ABC) transporters and has broad applicability to other classes of membrane proteins.
Pipeline
Our pipeline of PFMs holds the promise of restoring and enhancing membrane protein function linked to a wide array of diseases, offering renewed hope to patients. Our initial approach focuses on ABC transporter-mediated diseases.
Family
selection
Phase 1
ABC transporter
Expansion indications:
PBC, LPAC, GACC, PFICs, and NASH
selection
Phase 1
ABC transporters
selection
Phase 1
ABC transporters
selection
Phase 1
(ABC, SLC, GPCR, and ion channel)
selection
Phase 1
PSC, primary sclerosing cholangitis; PBC, primary biliary cholangitis; LPAC, low phospholipid associated cholelithiasis; GACC, genetically associated chronic cholestasis; PFIC, progressive familial intrahepatic cholestasis; NASH, nonalcoholic steatohepatitis; CKD, chronic kidney disease; AVS, aortic valve stenosis; AMN, adrenomyeloneuropathy; ALD, adrenoleukodystrophy
Our lead candidate, RTY-694
Our lead candidate, RTY-694, is an orally available dual-acting Positive Functional Modulator. RTY-694 improves bile composition and enhances bile flow. This novel and differentiated mechanism of action addresses both cholestasis and cholangitis associated with multiple hepatobiliary diseases. RTY-694 is currently in IND-enabling studies, with primary sclerosing cholangitis (PSC) as our initial indication.
PSC is a debilitating orphan bile duct disease characterized by inflammation, fibrosis, and narrowing of the medium and large bile ducts in the liver. Patients living with PSC experience a high symptom burden that reduces their quality of life and often requires hospitalization. Nearly half of PSC patients will eventually require liver transplant due to disease progression and complications and are at a 400-fold increased annual risk of cholangiocarcinoma versus the general population. RTY-694 neutralizes bile acids and free cholesterol and reduces bile stasis which directly addresses a core mechanism of PSC disease progression.
Our cardio-renal-metabolic programs
Poor cardio-renal-metabolic (CRM) health is a leading cause of death worldwide and a significant burden to the entire healthcare system. CRM health impacts nearly every organ system, and Rectify is advancing multiple cardio-renal-metabolic programs to address this group of interconnected disorders, including vascular calcification, aortic valve stenosis, and chronic kidney disease.
CKD and CKD-related vascular diseases are a leading cause of morbidity and mortality. Cardiovascular complications are projected to result in over $100 billion in direct costs in the U.S. in 2027. Our ABCC6-targeted PFM addresses a root cause of CKD-related vascular disease, vascular calcification. Inorganic pyrophosphate (PPi) is a potent anti-calcific factor, and CKD patients have low levels of circulating PPi. ABCC6 accounts for ~60% of all plasma PPi, Rectify’s ABCC6 PFM increases plasma PPi, reduces vascular calcification, and improves kidney function in a severe model of chronic kidney disease.
Team
Leadership
Board of Directors
Scientific Advisory Board
Publications
Explore the research behind our Positive Functional Modulator (PFM) therapies.