X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.
Etherna Immunotherapies NV has reported a milestone in its ongoing partnership with Dropshot Therapeutics Inc. Based on Etherna’s nucleic acid and lipid nanoparticle (LNP) capabilities, Dropshot has elected to advance one of its selected targets in renal disease to the clinical stage.
A Convalife Pharmaceuticals Co. Ltd. and Zhejiang Convalife Pharmaceutical Co. Ltd. patent describes new complement factor B (CFB) inhibitors potentially useful for the treatment of IgA nephropathy, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, age-related macular degeneration, cardiovascular disorder and cancer.
Small noncoding RNAs (sncRNAs), including miRNAs, piRNAs and snoRNAs, can provide further biological insights into the mechanisms of chronic kidney disease (CKD) in diabetes. Researchers from Leiden University Medical Center and collaborating institutions previously discovered that various classes of circulating sncRNAs are associated with kidney function (eGFR, uACR) and prevalent diabetic CKD.
Acute kidney injury (AKI), although sometimes reversible, is associated with an increased risk of chronic kidney disease (CKD) and progression to end-stage renal disease (ESRD). Reactive oxygen species (ROS) contribute to maladaptive repair and progression to CKD.
Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint.
Maze Therapeutics Inc. continues its journey toward a pivotal program after sharing positive top-line data from the phase II Horizon study with MZE-829, an oral, small-molecule, dual-mechanism APOL1 inhibitor, in patients with broad APOL1-mediated kidney disease (AMKD).
Vertex Pharmaceuticals Inc. is on track to complete a BLA by the end of March seeking potential U.S. accelerated approval for povetacicept, its dual inhibitor of the BAFF and APRIL cytokines, to treat immunoglobulin A nephropathy (IgAN), following a successful phase III Rainier trial.
Frontier Biotechnologies Inc. has entered into an exclusive licensing agreement with GSK plc whereby GSK will obtain exclusive worldwide rights to develop, manufacture and commercialize two of Frontier’s small interfering RNA (siRNA) pipeline products.
Asahi Kasei Pharma Corp. has advanced its novel endothelin ETA receptor antagonist AK-1960 into phase I in Japan for the treatment of various refractory diseases, such as chronic kidney disease.
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