Orphagen Pharmaceuticals

About Us


Orphagen Pharmaceuticals is a privately owned, emerging pharmaceutical company located in San Diego, California.


We focus on discovering new classes of small molecule drugs to previously unexplored drug targets from the nuclear receptor family. Our goal is to commercialize novel drugs to treat diverse types of serious disease.


Orphagen's first-to-ligand approach to discovery for orphan nuclear receptors addresses the pharmaceutical industry's need for innovative products. This approach also reduces the risks of downstream drug development by providing early characterization of the first pharmacologically active molecules in cellular and animal models of disease.


Our technology and expertise allow us to rapidly discover and validate ligands for previously unexplored orphan members of the nuclear receptor family. These are diverse targets, and our active programs span a range of therapeutic areas, including autoimmune disease, oncology, ophthalmological disease, and CNS disorders. This approach has already yielded specific ligands for several targets.


We intend to work with partners to pursue further development of our programs. Our first partnership was established with Japan Tobacco to discover, develop and commercialize drugs in the areas of immunology and autoimmunity.


Orphagen Pharmaceuticals has been funded through partnership revenues and $5.2 million in external grant funding.