ABCC6 positive functional modulator

Rectify Pharmaceuticals is developing oral small molecule positive functional modulators (PFMs) targeting the ABCC6 (ATP-binding cassette sub-family C member 6) transporter. ABCC6 is primarily expressed in the liver and is responsible for the efflux of ATP into the vasculature, where it is converted into inorganic pyrophosphate (PPi), a potent inhibitor of ectopic calcification. Mutations or dysfunction in ABCC6 lead to low systemic PPi levels, resulting in pathological mineralization. This program specifically targets vascular calcification associated with Chronic Kidney Disease (CKD), where ABCC6 activity may be compromised. By restoring or enhancing ABCC6 function, the therapy aims to increase systemic PPi levels and prevent or treat pathological vascular mineralization. The program is currently in the preclinical stage of development.

Other names

ABCC6 positive functional modulator (Rectify Pharmaceutical)Rectify ABCC6 program