AAV9-coGBA1.1

AAV9-coGBA1.1 is an adeno-associated virus serotype 9 (AAV9) gene therapy designed to treat neuronopathic Gaucher disease (nGD) and GBA1-associated Parkinson disease. Developed by researchers at the National Human Genome Research Institute (NHGRI), the vector carries a codon-optimized human GBA1 transgene (coGBA1.1) under the control of the EF1s promoter, stabilized by the hepatitis B virus post-transcriptional regulatory element (HPRE). The therapy aims to restore glucocerebrosidase (GCase) activity in the central nervous system, addressing the blood-brain barrier limitations of current enzyme replacement therapies. In preclinical studies, systemic administration has demonstrated the ability to prevent early lethality in GD2 mouse models and extend lifespan in GD3 models, while also restoring GCase activity in human iPSC-derived dopaminergic neurons.