Vertex Pharmaceuticals Inc. has described polycystin-1 (PKD1) (mutant) correctors reported to be useful for the treatment of autosomal dominant polycystic kidney disease.
Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. has synthesized cytochrome P450 11B2, mitochondrial (CYP11B2; aldosterone synthase; ALDOS) inhibitors reported to be useful for the treatment of hypertension, diabetic nephropathy, chronic kidney disease and primary aldosteronism.
Researchers have discovered that reduced expression of the free fatty acid receptor 4 (FFAR4) is a biomarker of podocyte injury and aging, as well as a therapeutic target. Podocyte injury leads to progression of glomerular disease and aging, but the underlying mechanisms are poorly understood.
With a 42% placebo-adjusted reduction in proteinuria at week 36, Vera Therapeutics Inc. met its primary endpoint in the Origin phase III trial with BAFF and APRIL dual inhibitor atacicept in treating immunoglobulin A nephropathy (IgAN) in adults.
Suzhou Sanegene Bio Inc. has gained clinical trial approval in China for SGB-3383 for the treatment of complement-mediated kidney diseases, including IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis and atypical hemolytic uremic syndrome.
Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
Each year, acute kidney injury affects more than 13 million people and leads to nearly 2 million deaths. It can occur, for example, in cancer patients taking nephrotoxic cisplatin chemotherapy and in individuals who suffer sepsis or ischemic stroke.
Scientists at Jiangsu Hengrui Medicine Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have identified relaxin receptor 1 (RXFP1; LGR7) agonists reported to be useful for the treatment of heart failure, chronic kidney disease, acute kidney injury, fibrosis and inflammatory disorders.