Dent disease (DD) is an X-linked recessive disorder characterized by low molecular weight proteinuria (LMWP), kidney stones and progressive kidney failure.

OTU deubiquitinase 5 (OTUD5) is a deubiquitinating enzyme that has been shown to play a key role in mediating innate immunity and inflammation development, and which is also involved in various cancers.

Eleva is preparing a first clinical study in this indication, with dosing slated to begin in in the first half of next year.

Previous studies have demonstrated that CaMKKβ/AMPK signaling plays an important role in regulating mitochondrial homeostasis and that down-regulation of CaMKKβ could contribute to the pathogenesis of diabetic kidney disease (DKD).

Researchers from Purespring Therapeutics Ltd. and affiliated organizations have presented preclinical data for the adeno-associated vector (AAV) gene therapy PS-001 for the treatment of glomerular disease.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure characterized by development of multiple fluid-filled cysts linked to increased cAMP levels and fibrosis in the kidneys leading to progressive renal failure and need for dialysis or renal transplant.

Podocytes are a terminally differentiated cell type located in the glomerulus. Podocyte damage and the subsequent dysregulation of podocyte proteins have been implicated in various kidney disorders. Since gene delivery to podocytes using adeno associated vectors (AAVs) has been challenging due to various technological and physiological hurdles, investigators at Purespring Therapeutics Ltd. developed an AAV gene therapy platform that allowed for effective, specific and safe delivery of transgenes to podocytes.