Tunable epileptic gene therapy / CODA Biotherap - LARVOL DELTA

Assessment of AAV Regulatory Cassettes with Optimal Hippocampal Neuron Expression for the Treatment of Focal Epilepsy (ASGCT 2022) - "To create an effective gene therapy approach for focal epilepsy, we generated a series of AAV expression cassettes that could drive optimal expression of our LGIC in target cells of the hippocampus...mRNA analysis using ddPCR and immunofluorescence for cellular tropism demonstrated high levels of expression from human versions of the Syn- and CaMKII-driven cassettes. Further assessment of the two selected cassettes, one driven by the pan-neuronal hSyn promoter and the other by CaMKII, an excitatory cell-specific promoter, will be performed in an animal model of focal epilepsy to finalize the development candidate."

CNS Disorders • Epilepsy • Gene Therapies • Neuroblastoma • Oncology • Solid Tumor