coAAV-CSF-01

coAAV-CSF-01 is a novel, central nervous system (CNS)-targeted gene therapy vector developed by Coave Therapeutics using its proprietary ALIGATER platform. This vector is based on an engineered adeno-associated virus serotype 2 (AAV2) capsid that has been chemically conjugated with targeting ligands to enhance tissue specificity and transduction efficiency. In preclinical studies in non-human primates, coAAV-CSF-01 demonstrated dramatically improved CNS delivery compared to AAV9, with up to 100-fold higher transgene expression in the cortex and 10,000-fold higher expression in the hippocampus at equivalent doses. The vector achieves robust brain transduction via intra-cerebrospinal fluid administration while significantly reducing peripheral organ exposure—including strong liver de-targeting—thereby addressing key safety concerns associated with traditional AAV vectors. The ALIGATER platform enables modular modification of AAV capsids for optimized tissue targeting and therapeutic performance, supporting the development of genetic medicines for neurodegenerative and neurodevelopmental CNS disorders[1][2][5][6].