Rectifying function, Restoring hope
See how we are applying our PFM product platform to rectify the 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 site of the protein’s function––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 targeting this dysfunction 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 has 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 new 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 Hepatobiliary program
Our lead program is focused on developing an orally available dual-targeted ABCB4 and BSEP PFM that improves bile composition and enhances bile flow. This novel and differentiated mechanism of action addresses both cholestasis and cholangitis associated with multiple hepatobiliary diseases. Our lead candidate is currently in IND-enabling studies with our initial indication being primary sclerosing cholangitis (PSC).
PSC is a debilitating orphan disease characterized by inflammation, fibrosis, and narrowing of the medium and large bile ducts in the liver. Patients living with PSC must endure 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. PSC patients are at a 400-fold increased annual risk of cholangiocarcinoma versus the general population. Our dual-targeted ABCB4 and BSEP PFMs act to neutralize bile acids and free cholesterol and reduce bile stasis, directly addressing 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.