Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a genetic liver disorder caused by mutations in the ABCB4 gene encoding multidrug resistance protein 3 (MCP3) in humans, a biliary phospholipid transporter. Rectify Pharmaceuticals Inc. has developed the novel compound RTY-694, a dual-acting MDR3/BSEP positive modulator that increased the protein function of both MDR3 and BSEP.
The ionic and metabolic impairment observed in chronic kidney disease (CKD) leads to vascular calcification, which can induce cardiovascular events and mortality. Several factors may impact the progression of vascular calcification, where inorganic pyrophosphate plays a crucial inhibitory role.
Rectify Pharmaceuticals Inc. has conducted preclinical testing on RTY-694, an oral dual-targeted positive functional modulator for use in the treatment of hepatobiliary disorders.
Rectify Pharmaceuticals Inc. has nominated the positive functional modulator (PFM) RTY-694 as a development candidate for primary sclerosing cholangitis (PSC). As the lead PFM in Rectify’s hepatobiliary program, RTY-694 is now advancing to first-in-human trials for PSC.
Jonathan Moore, who was employee No. 36 when he came to Vertex Inc. in 1990 and ended up spending the next 28 years at the company, realized he wasn’t done with his work in ATP-binding cassette (ABC) transporters and has now formed Rectify Pharmaceuticals Inc. The new company just closed on a $100 million series A financing to fund development of a pipeline of therapies to restore ABC transporter function for treating genetic diseases.
