SmartPharm Therapeutics, now a subsidiary of Sorrento Therapeutics, is a gene therapy company applying the next generation of non-viral DNA and RNA technologies to treat serious human diseases. Founded with the vision of producing “Biologics from Within,” SmartPharm is focused on bringing together unique technology and developmental capabilities for the treatment of rare diseases including lysosomal storage disorders. SmartPharm Therapeutics is a second-generation gene-encoded therapeutics company that has benefited from the first decades of learning in gene therapy development to produce an evolved therapeutic platform. We integrate these insights with key technologies and engineering capabilities to enable us to produce optimal gene-encoded therapeutics for patients.