AAVrh10-coGBA1.1

AAVrh10-coGBA1.1 is an adeno-associated virus (AAV) serotype rh10-based gene therapy candidate developed for the treatment of neuronopathic Gaucher disease (nGD) and GBA1-associated Parkinson disease. The vector utilizes a codon-optimized human GBA1 transgene (coGBA1.1) under the control of an EF1s promoter and is stabilized by a hepatitis B virus post-transcriptional regulatory element (HPRE). By delivering a functional GBA1 gene, the therapy aims to restore glucocerebrosidase (GCase) enzyme activity, thereby mitigating the accumulation of glucosylceramide and neuroinflammation. Preclinical studies conducted by the National Human Genome Research Institute (NHGRI) demonstrated that systemic administration of AAVrh10-coGBA1.1 can restore GCase levels and extend lifespan in murine models of Gaucher disease type 2, although it showed lower durability compared to an AAV9-based counterpart.