Translating genetic discoveries into cures is rapidly becoming reality
Once delivered, the gene and promoter, which activates the gene, may lead to therapeutic protein expression. With gene therapy, the delivered gene does not integrate into the genome, so this approach can be curative in slow- or non-dividing cells (e.g., adult liver or central nervous system).
Our unique vectors have demonstrated significant systemic biodistribution to multiple tissue types in preclinical studies, including liver, central nervous system (CNS), muscle (skeletal and cardiac) and eye. This enables us to potentially address a broad range of genetic disorders.