Scholars

Twenty scientists have been selected for OHC’s 2024 and 2025 Rare Disease Scholar Award to advance academic discoveries into clinical practice for rare diseases.

2025 OHC Rare Disease Scholars

Rachel Bailey, PhD

Rachel Bailey, PhD

University of Texas Southwestern

Gene Therapy to Cure an Ultra-Rare, Devastating Neurodevelopmental Metabolic Disorder of the SLC13A5 Citrate Transporter

Esther Becker, PhD

University of Oxford

New Small Molecules to Treat Spinocerebellar Ataxia

Joseph Buxbaum, PhD

Joseph Buxbaum, PhD

Icahn School of Medicine

Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome with Small Molecule Drugs

Matthew Gentry, PhD

Matthew Gentry, PhD

University of Florida

Advanced Brain Penetrant Enzyme Therapy to Treat Neurological Glycogen Storage Diseases

Michael Lin, MD, PhD

Michael Lin, MD, PhD

Stanford University

A Genetically Programmed Therapeutic for Sensing and Killing Rare Brain Cancers

Pengfei Liu, PhD

Pengfei Liu, PhD

Baylor College of Medicine

Precision Nucleic Acid Therapeutics for the Ultra-Rare CAMK2B Neurodevelopmental Disorder

David Segal, PhD

David Segal, PhD

University of California, Davis

Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome with Nucleic Acid Drugs

Anthony Shum, MD

Anthony Shum, MD

University of California, San Francisco

Gene Therapy for the Progressive, Debilitating, Multisystem COPA Syndrome

Albert La Spada, MD, PhD

Albert La Spada, MD, PhD

University of California, Irvine

Silencer RNA Therapy to Treat Rare, Juvenile-onset ALS Neuromuscular Disease, ALS4

Mingshan Xue, PhD

Mingshan Xue, PhD

Baylor College of Medicine

Gene Therapy for the Catastrophic CASK-Related Neurodevelopmental Disorder

2024 OHC Rare Disease Scholars

Jacquelyn Bower, PhD

Jacquelyn Bower, PhD

University of North Carolina at Chapel Hill

Setting Sights on Gene Therapy to Treat a Deadly Eye Cancer

Louis Chesler, MD, PhD

Louis Chesler, MD, PhD

Centre for Pediatric Oncology Experimental Medicine, The Institute of Cancer Research

Flipping a switch with CAR-T cells

Charles Gersbach, PhD

Charles Gersbach, PhD

Duke Center for Advanced Genomic Technologies

Stopping Hodgkin lymphoma with supercharged T cells

Xianxin Hua, MD, PhD

Xianxin Hua, MD, PhD

University of Pennsylvania

Redefining CAR T Therapy for Acute Myeloid Leukemia

Michele Jacob, PhD

Michele Jacob, PhD

Dept. of Neuroscience Tufts University School of Medicine

Hope for Children with CTNNB1 Syndrome

Bowen Li, PhD

Bowen Li, PhD

University of Toronto

Deploying Precision Medicine with Nanotechnology to Treat Cystic Fibrosis

Michael Pacold, MD, PhD

Michael Pacold, MD, PhD

NYU Langone Health

Rescue for HPDL-Driven Mitochondrial Disorders

Carlo Rinaldi, MD PhD

Carlo Rinaldi, MD PhD

University of Oxford

Turning the Switch on Spinal and Bulbar Muscular Atrophy

Timothy Yu, MD, PhD

Timothy Yu, MD, PhD

Boston Children’s Hospital

A Pivotal Innovation Brings New Hope

Haiyan Zhou, MD, PhD

Haiyan Zhou, MD, PhD

University College London

Nucleic Acid Therapy to Treat a Rare Neuropathy

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