Diabetic kidney disease (DKD) is a chronic complication of type 1 and type 2 diabetes with albuminuria and progressive kidney failure; about 20%-40% of patients with diabetes will develop some type of kidney disease.
IgA nephropathy is a progressive kidney disease characterized by the deposition of immune complexes containing IgA, where a proliferation-inducing ligand (APRIL) drives the production of pathogenic IgA. Jade Biosciences Inc. has developed a novel APRIL-binding therapeutic, JADE-101, for the treatment of IgA nephropathy.
Morphic Therapeutic Inc. has identified compounds acting as integrin αvβ1 antagonists reported to be useful for the treatment of cancer, chronic kidney disease, diabetic nephropathy, pulmonary arterial hypertension, idiopathic pulmonary fibrosis, interstitial lung diseases, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH) and primary biliary cholangitis.
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.
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.
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.
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