AAV9-JGA

AAV9-JGA is a novel adeno-associated virus (AAV)-mediated gene therapy vector engineered for highly specific targeting of CLDN16/SLC12A1-positive thick ascending limb (TAL) cells within the juxtaglomerular apparatus (JGA) of the kidney. Developed through an in vivo selection strategy from a liver-detargeting AAV9 peptide display library, this vector demonstrates superior specificity compared to wild-type AAV9. It accesses target cells directly via the tubule-afferent arteriole contact, bypassing the glomerular filtration barrier. AAV9-JGA has shown the capacity to modulate renal function through targeted cell ablation (when expressing diphtheria toxin receptor) and to deliver therapeutic proteins like uromodulin into both urine and bloodstream, offering promising avenues for targeted therapies in chronic kidney disease. Its performance is currently being evaluated in large animal models.