2024 Million Dollar Bike Ride Pilot Grant Program
The Orphan Disease Center (ODC) at the University of Pennsylvania is pleased to announce the 2024 Million Dollar Bike Ride Pilot Grant Program is now open and offering up to 42 different research grant opportunities focusing on 34 different rare diseases.
This program provides a one‐year grant to support research related to a rare disease represented in the 2024 Million Dollar Bike Ride. The number of awards and dollar amounts vary per disease based on fundraising totals by each disease team. This Request for Applications (RFA) is open to the international research community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.
One $70,000 grant is available for Schinzel-Giedion Syndrome (SETBP1 gain of function) research.
The Schinzel-Giedion Syndrome Foundation is seeking research proposals that will advance the understanding of disease mechanisms and pre-clinical discovery and/or development of potential therapies and tools. Areas of interest include, but are not limited to:
- Discovery and validation of biomarkers (molecular and functional). To date, no SGS-specific biomarkers have been identified.
- Disease models. The multisystemic nature of SGS may require multiple models of disease development and progression, including, but not limited to, brain, renal, and respiratory. The SGS community is interested in funding disease model development that may lead to the discovery of therapeutic strategies for intervention. High throughput-amenable models are encouraged.
- Novel therapeutic approaches for SGS. The multisystemic nature of symptoms requires the development of multiple therapeutic treatments for the SGS community. Approaches we are interested in funding include (but are not limited to): drug repurposing, small molecules, PROTAC, gene therapies, and RNA-based therapies.
- Identification of disease mechanisms. Developing specific treatments highly depends on understanding how variants impact protein function and lead to disease phenotypes. We are interested in exploring new areas of research which we feel have huge translational relevance to SGS, such as the role of immune dysfunction.
Applicants are encouraged to collaborate with existing SGS researchers and to leverage existing disease models and data (animal models, patient-derived cell models in our CombinedBrain biobank, clinical health data in our patient registry on AcrossHealthcare’s Matrix platform), and to contact the Schinzel-Giedion Syndrome Foundation at contact@sgsfoundation.org with any questions about these resources.
This grant is made possible by Team SETBP1Strong and The Schinzel-Giedion Syndrome Foundation. To find out more please visit the ODC website
