Scholars
Ten scientists have been selected for OHC’s Inaugural 2024 Rare Disease Scholar Award to advance academic discoveries into clinical practice for rare diseases.
Jacquelyn Bower, PhD – University of North Carolina at Chapel Hill
A novel adeno-associated virus (AAV) gene therapy approach for the treatment of rare Uveal Melanoma Tumors
Louis Chesler, MD, PhD – Institute of Cancer Research
Engineering chimeric antigen receptors fused to a high-efficiency lenalidomide-degradable Tag to control CAR-T cell activity - iTAG3
Charles Gersbach, PhD – Duke University
Epigenetically enhanced cell therapy for Hodgkin's Lymphoma
Xianxin Hua, MD, PhD – University of Pennsylvania
A new cellular strategy for treating acute myeloid leukaemia
Michele Jacob, PhD – Tufts University School of Medicine
A drug to treat brain abnormalities caused by CTNNB1 defects, an ultra-rare disease
Bowen Li, PhD – University of Toronto
A nucleic acid therapy to treat cystic fibrosis, an inherited lung disease
Michael Pacold, MD, PhD – New York University
Drugs restoring brain function in rare metabolic diseases
Carlo Rinaldi, MA, MD, PhD – University of Oxford
Nucleic acid therapy for spinal and bulbar muscular atrophy (SBMA), a rare X-linked neuromuscular condition
Timothy Yu, MD, PhD – Boston Children’s Hospital
Custom DNA-based therapy for a rare genetic disorder (PEX1 Zellweger Syndrome)
Haiyan Zhou, MD, PhD – University College London
Nucleic acid therapy to treat loss of sensation and paralysis caused by a rare mutation