AAV-DB-3

AAV-DB-3 is a novel adeno-associated virus (AAV) capsid variant engineered for highly efficient gene delivery to deep brain structures, particularly the basal ganglia and associated cortical regions. Developed by Latus Bio in collaboration with academic researchers, including those at Children's Hospital of Philadelphia (CHOP), AAV-DB-3 was identified through a large-scale screen of millions of AAV variants in non-human primates. It demonstrates superior potency compared to existing clinical vectors, achieving over 180-fold higher transgene mRNA expression in Huntington’s disease-relevant brain regions than wild-type AAV5 at equivalent doses. The vector specifically targets medium spiny neurons of the striatum and projection neurons of the cortex—key cell types affected in neurodegenerative diseases such as Huntington’s disease and Parkinson’s disease. In preclinical studies using mice, non-human primates, and human induced pluripotent stem cell-derived neurons, AAV-DB-3 enabled robust gene transfer at doses 10–100 times lower than current clinical standards. Its enhanced tropism for human neurons supports its potential for future clinical translation as a one-time precision therapy for neurodegenerative disorders[1][4][5][8].