Scholars

Rachel Bailey, PhD – University of Texas 

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 – Icahn School of Medicine

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

Matthew Gentry, PhD – University of Florida

Advanced Brain Penetrant Enzyme Therapy to Treat Neurological Glycogen Storage Diseases

Albert La Spada, MD, PhD – University of California

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

Michael Lin, MD, PhD – Stanford University

A Genetically Programmed Therapeutic for Sensing and Killing Rare Brain Cancers

Pengfei Liu, PhD – Baylor College of Medicine 

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

David Segal, PhD – University of California

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

Anthony Shum, MD – University of California

Gene Therapy for the Progressive, Debilitating, Multisystem COPA Syndrome

Mingshan Xue, PhD – Baylor College of Medicine

Gene Therapy for the Catastrophic CASK-Related Neurodevelopmental Disorder

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

Bowen Li, PhD – University of Toronto 

A nucleic acid therapy to treat cystic fibrosis, an inherited lung disease 

Michele Jacob, PhD – Tufts University School of Medicine

A drug to treat brain abnormalities caused by CTNNB1 defects, an ultra-rare disease

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)

Michael Pacold, MD, PhD – New York University

Drugs restoring brain function in rare metabolic diseases 

Haiyan Zhou, MD, PhD – University College London

Nucleic acid therapy to treat loss of sensation and paralysis caused by a rare mutation