Orphagen Pharmaceuticals
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Orphagen is a pioneer in the screening, discovery and development of ligands that modulate orphan or unexplored members of the nuclear receptor family. We have proprietary leads for novel drug targets with potential applications in precision oncology and autoimmune disease. Our scientists and management team are committed to the best we can bring to bear in pursuit of new therapies.

We are drug hunters. Our mission is translational drug discovery and development with novel targets leading to first-in-class industry partnerships.
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We are committed to creating innovative science for discovery of new therapies at novel drug targets. He has 30 years of experience in life sciences research and pharmaceutical R&D. Scott led Orphagen to its first partnership in 2008 with JT Pharma and created first-in-class discovery programs for two other orphan nuclear receptors, steroidogenic factor-1 (SF-1) and ROR-beta.
Orphagen's proprietary screening technology has led to successful discovery of the first drug-like small molecules to several orphan nuclear receptors. In addition, Orphagen performs critical target validation in animal and cellular models of disease based on its novel, potent and specific small molecule ligands.
Adrenocortical carcinoma (ACC) is a rare cancer of the adrenal gland with an annual incidence of 0.7-2.0 per million population. Peak incidence occurs in the fourth decade of life. Surgical resection of the tumor is the mainstay of treatment with addition of adjuvant chemotherapy. Even with complete resection, the rate of recurrence ranges from 20% to 35% in those patients with no residual disease after surgery.
Orphagen's strength is drug discovery at novel, unexplored nuclear receptors. Our discovery objective has been to validate new therapies at these targets in animal models of disease with our potent receptor ligands. Two programs are in the discovery phase at two undisclosed targets. In each case, we have potent and unique ligands-new chemical entities-to use as tool for potential development compounds.
Orphagen is an industry leader in orphan nuclear receptor translational research. We have developed and validated transcriptional and biochemical assays for a panel of nuclear receptors. We provide flexible nuclear receptor profiling and screening services. Compounds can be screened against single or multiple receptors in single or multiple doses for selectivity profiling and/or IC50 determination.
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