SMARTPHARM THERAPEUTICS, INC

Capabilities

SmartPharm Therapeutics is a Massachusetts based, next-generation nonviral gene therapy company. Our technology platform has the potential to address a wide range of serious and rare diseases (“biologics from within”). We use either DNA or mRNA along with advanced nanoparticle delivery methods to create gene-encoded therapeutics. Our first product is a DNA-based enzyme replacement therapy for Gaucher disease, a rare inherited genetic disorder. Our goal is to become an clinical stage therapeutics company that will generate a series of NDA-stage gene-encoded therapies for licensing, partnering, or acquisition. We would likely seek to commercialize in partnership or utilizing an outsourced model.