Awarded Grants

The ODC offers over 50 grant opportunities in 30+ disease areas annually to researchers across the globe, as well as within the Penn and CHOP communities. Since 2011, our grant programs have funded $17.2 million in rare disease research.

See below for a complete list of ODC-awarded grants since 2011.

Click HERE for the full list of 2018 Million Dollar Bike Ride Pilot Grant Awardees!

Awarded Grants

Displaying Results 1 - 10 of 216

Awardee Project Title Institution Grant Amount Funding Year
Jamie Fitzgerald, PhD A novel RNA-based CRISPR strategy for treating congenital muscular dystrophy Henry Ford Health System MDBR - Congenital Muscular Dystrophy $50,699 2019
Brian Bigger, PhD Targeted stem cell gene therapy for severe Mucopolysaccharidosis I (Hurler) University of Manchester MPS I Pilot Grant Program $165,000 2019
Natalia Gomez-Ospina, MD Optimizing cell selection and conditioning regimens for autologous transplantation of human genome-edited hematopoietic stem cells to treat MSPI Stanford University MPS I Pilot Grant Program $165,000 2019
Juliette Hordeaux, DVM, PhD Engineering AAV vector genome to evade immune responses and associated toxicity after intrathecal AAV gene therapy University of Pennsylvania MPS I Pilot Grant Program $165,000 2019
Edward Gilmore, MD, PhD Therapeutic screen for DNA damage in A-T patient derived motor neurons. Case Western Reserve University School of Medicine MDBR - Ataxia-Telangiectasia $87,164 2019
M. Leonor Cancela, PhD Zebrafish as model to analyze CDKL5 function University of Algarve MDBR - CDKL5 $51,382 2019
Hong Zhang, PhD Mechanistic study of WDR45/45B and their binding partner ATG2 in the autophagy pathway of neural cells University of Massachusetts Medical School MDBR - A Neurodegeneration with Brain Iron Accumulation Disorder $51,020 2019
Robin Ketteler, PhD. The use of a patient-centric neuronal cell model of Beta-Propeller Protein-Associated Neurodegeneration (BPAN) as a platform to develop novel therapies University College London MDBR - BPAN-A Neurodegeneration with Brain Iron Accumulation Disorder $51,020 2019
Tommaso Pizzorusso, PhD Validation of pupil size as a biomarker for CDKL5 disorder: longitudinal assessment and relationship with disease severity. University of Florence MDBR - CDKL5 $51,382 2019
Nigel Farrow, PhD Airway Cell Therapy for Cystic Fibrosis Nonsense Mutations University of Adelaide MDBR - Cystic Fibrosis $53,668 2019
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