Chief Scientific Officer, Ascendis Pharma
TransCon technology for sustained delivery of proteins, peptides, and small molecules
Peptide and protein drug candidates often suffer from suboptimal properties such as poor bioavailability and inadequate circulation half-life due to rapid renal clearance and/or receptor- mediated clearance. The TransCon technology enables transient conjugation of a parent drug to a carrier through a linker to create a polymeric prodrug. Following injection, the proprietary linker autohydrolyzes in an enzyme-independent process based on pH and temperature, and unmodified parent drug is released at a rate fine-tuned through the design of the TransCon linker. Since drug is released in its native form, its original pharmacology is retained, with the same biodistribution, receptor affinity, and mode-of-action as the parent drug. Linkers have been developed with release rates that allow for the development of prodrugs that can deliver proteins, peptides, and small molecules with dosing frequencies ranging from days to several months. Soluble as well as insoluble carrier systems can be used to enable systemic or localized delivery. The TransCon technology has been used to develop a pipeline of rare endocrinology disease programs: TransCon Growth Hormone (in phase 3 for pediatric growth hormone deficiency), a long-acting prodrug of parathyroid hormone, TransCon PTH (in phase 1 for hypoparathyroidism), and a long-acting prodrug of C-type natriuretic peptide, TransCon CNP, (filed for initiation of clinical development in Australia for achondroplasia).
Harald Rau has served as the Chief Scientific Officer and SVP, managing the research group at Ascendis Pharma since December 2007. Prior to Ascendis Pharma, Dr. Rau served as the Chief Scientific Officer of Complex Biosystems GmbH, a biotechnology company he co-founded in 2002, which was acquired by Ascendis Pharma in December 2007. Prior to co-founding Complex Biosystems, he worked with Graffinity Pharmaceuticals, a biotechnology company, from 1998 to 2002. Dr. Rau holds a doctorate from the University of Freiburg, Germany.