CTx-GBA1

CTx-GBA1 is an adeno-associated virus (AAV)-based gene therapy being developed by Coave Therapeutics using its AAV-ligand conjugate (coAAV) capsid platform to treat Parkinson’s disease and other disorders associated with GBA1 mutations by restoring glucocerebrosidase (GCase) activity. The investigational therapy delivers a functional GBA1 transgene to the brain, where intrastriatal administration of the coAAV2-GBA1 construct in preclinical models leads to dose-dependent restoration of GCase activity, reduction of glycolipid substrate accumulation, and improvement of lysosomal function.[1][11][17][6] Coave is advancing CTx-GBA1 as a preclinical program in rare CNS disease and GBA1-associated Parkinson’s disease, aiming to provide a disease-modifying approach by correcting the underlying lysosomal enzyme deficiency rather than only treating motor symptoms.[1][11][17][6]