SmartPharm 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 treatment of rare diseases through enzyme replacement.
SmartPharm is focused on transforming biologics therapy in high-revenue markets. Our lead program addresses the replacement of an enzyme to alleviate Gaucher disease, a serious orphan disease. The ability to address Gaucher disease would be the first SmartPharm target in a group of rare diseases known as lysosomal storage disease that include Fabry and Pompe disease and represent a large potential pipeline for the technology. In addition, our platform includes our RNA programs that address tissue regeneration/restoration following acute myocardial infarction or bone marrow transplantation.