Virtual R&D Event: Rectifying Protein Dysfunction: RTY-694 for the Treatment of Primary Sclerosing Cholangitis
Thursday, November 21, 11:00am – 1:00pm EST

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.

Positive Functional Modulators - about section graphic
PFM Product Platform Image

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.

Therapeutic area
Target(s)
Family
Indications
Discovery
Candidate
selection
IND-enabling
Clinical
Phase 1
Hepatobiliary
Dual targeted ABCB4/BSEP
ABC transporter
Lead indication: PSC
Expansion indications:
PBC, LPAC, GACC, PFICs, and NASH
Discovery
Candidate
selection
IND-enabling
Clinical
Phase 1
Cardio-renal-metabolic
ABCC6
ABC transporters
CKD, AVS
Discovery
Candidate
selection
IND-enabling
Clinical
Phase 1
Neuro
Dual targeted ABCD1/ABCD2
ABC transporters
AMN, ALD
Discovery
Candidate
selection
IND-enabling
Clinical
Phase 1
Additional
Multiple
(ABC, SLC, GPCR, and ion channel)
Diseases caused by protein dysfunction
Discovery
Candidate
selection
IND-enabling
Clinical
Phase 1
RTY-694, is an orally available dual-acting Positive Functional Modulator.

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.

cardio-renal-metabolic image

Team

Leadership

Rajesh Devraj, Ph.D.
President and Chief Executive Officer
Sachiyo Minegishi
Chief Operating Officer
Pol Boudes, M.D.
Chief Medical Officer
Bharat Reddy, Ph.D.
Chief Business Officer
Robert Hughes, Ph.D.
Executive Vice President, Drug Discovery and Preclinical Development
John Miller, Ph.D.
Vice President, Head of Biology

Board of Directors

General Partner, Forbion
President and Chief Executive Officer
Venture Partner, Longwood Fund
Chief Executive Officer, Q32 Bio
Chief Executive Officer, Ring Therapeutics
Executive Chair of the Board, Partner, Atlas Venture
Founder and Managing Partner, Omega Funds

Scientific Advisory Board

Chief Medical Officer, Q32 Bio
Independent R&D Consultant
Co-Founder and CSO, Accent Therapeutics
Founder and Chief Science Advisor, Rectify Pharma
CEO Liver Consulting LLC
Professor of Molecular Hepatology, School of Immunology & Microbial Sciences at King’s College London
Professor, Department of Integrative Structural and Computational Biology, Scripps Research Institute

Publications

Explore the research behind our Positive Functional Modulator (PFM) therapies.

News

Read our most recent press releases and company updates.

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