Daiichi Sankyo Co. Ltd. and Kyoto Pharmaceutical Industries Ltd. have identified Kelch-like ECH-associated protein 1 (Keap1)/Nrf2 interaction inhibitors reported to be useful for the treatment of acute lung injury, chronic kidney disease, autism, Alzheimer's disease, dry eye, heart failure, psoriasis and Sjögren's syndrome, among others.
It has been previously demonstrated that the two coding variants in the APOL1 gene (G1 and G2) are associated with a greater risk of progressive, proteinuric kidney disease; however, there currently are no therapies to address the causal genetic drivers of this disease. Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of a novel small-molecule inhibitor of APOL1, MZ-302, and they evaluated its efficacy in a new transgenic model of APOL1-mediated kidney disease (AKD).
PYC Therapeutics Ltd. has announced the results of a study of PYC-003 conducted in human 3D models derived from patients with end-stage renal failure due to autosomal dominant polycystic kidney disease (PKD).
G1 and G2 genetic variants of the human APOL1 gene have been previously associated with an increased risk of developing chronic kidney diseases (CKD) in the African American population, and recent studies have shown that inhibition of APOL1 ion channel function could represent a novel therapeutic strategy for the treatment of patients with APOL1-like nephropathies.
Researchers from Wake Forest University, Charles University and affiliated organizations have found a link between mutations in the APOA4 gene and inherited kidney disease.
Researchers from Taisho Pharmaceutical Co. Ltd. have discovered new heparanase-1 (HPSE1) inhibitors for the treatment of cancer and proteinuric kidney diseases.
African Americans are 10 times more likely to develop chronic kidney disease (CKD) than Americans of European descent in part due to inheritance of one of two high-risk (HR) APOL1 variants (G1/G2, not G0) that are only present in people of West African or Caribbean ancestry. By contrast, these two APOL1 HR variants confer a beneficial resistance to otherwise lethal trypanosomiasis, which is caused by a pathogen endemic to West Africa, but still this ultimately results in a 4-fold increased risk for end-stage kidney disease (ESKD).
Scientists at Egenesis Inc. have transplanted kidneys from genome-edited pigs into cynomolgus monkeys that remained functional for long periods after transplantation. The monkeys, whose own kidneys were removed during the surgery, survived for a median of 176 days after receiving one pig kidney. Maximal survival was just over 2 years. The data were published today in Nature. Egenesis CEO Mike Curtis told reporters that the study has achieved the longest survival to date “using clinically translatable immunosuppression … longer survival has been achieved using really aggressive immunosuppression that really isn’t clinically translatable.”
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