Pipeline

SmartPharm’s goal is to replace recombinant protein therapeutics with gene-encoded therapeutics (GETs). Our company has selected three areas of focus and has initiated product development in each of these areas:

Enzyme Replacement Therapy: Initial focus on lysosomal storage diseases.
Preservation and restoration of cell and tissue viability: Prevention of cell death from medical conditions or interventions.
Cancer biologics: Development of gene-encoded “biobetters” to existing cancer biologic drugs, including therapeutic monoclonal antibodies.

SmartPharm’s initial focus is on development of a DNA-based glucocerebrosidase treatment for Gaucher disease type 1. It has also advanced an mRNA-based treatment to preserve cardiac tissue following acute myocardial infarction into advanced preclinical development.

Learn more about our areas of development