VESPA SCN1A gene therapy

VESPA SCN1A gene therapy is an investigational therapeutic approach developed by Tevard Biosciences to treat Dravet syndrome, a severe form of epilepsy caused by haploinsufficiency of the SCN1A gene. The VESPA (Voltage-gated Enhancement by Specific Phosphorothioate-modified Antimes) platform utilizes a novel mechanism involving tRNA-based modulation. Specifically, it employs engineered tRNAs to enhance the translation of the remaining functional SCN1A mRNA, thereby increasing the production of the Nav1.1 sodium channel protein. This approach aims to restore physiological levels of the protein in inhibitory interneurons, potentially reducing seizure frequency and addressing the underlying cause of the disease without the size constraints associated with traditional viral-mediated gene replacement.

Other names

VESPA SCN1A