Programs of Excellence
The Programs of Excellence (PoE) at the Orphan Disease Center seek to fill critical gaps in translational research.
Each PoE focuses on a disease or cluster of related diseases with significant unmet need, and in which common therapeutic platforms (i.e., therapeutic antibodies, stem cells, gene therapy, RNAi, genome editing, etc.) can be applied. The goal of each PoE is to achieve substantial treatment effects through the identification and targeting of tractable molecular targets. We prioritize research into restoration of function and/or stabilization of a degenerative process. Each of our PoEs has viable strategies for securing sustainable funding.
The ODC Programs of Excellence support new therapeutic development for a disease while also building a more integrated ecosystem to support drug development while meeting the unique needs of the patient community. Each of our POEs has a committed Director that oversees the progress of the program and highlights needs for the field. Through the POEs we assess the need for research tools (antibodies, cell lines, animal models, etc), translational capabilities, clinical development (i.e.patient registries and natural history, biomarkers, clinical endpoints, etc), and we work closely with patients and patient groups in each of these areas to ensure there is a thorough understanding of the disease and community needs.
Mucopolysaccharidoses
Mucopolysaccharidoses (MPSs) are a family of lysosomal storage diseases, each of which is caused by the absence of a critical enzyme needed for normal lysosomal function. These lysosomal enzymes are needed to degrade mucopolysaccharides, now called glycosaminoglycans (GAGs), as well as other cellular debris.
Pilot POE Grants
The ODC Pilot Programs of Excellence Grant helps Penn and CHOP investigators to engage rare disease communities (patients, clinicians, industry) and build appropriate resources across these communities to improve standards of care or therapeutic development.