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