Agnes Chen

Dr. Agnes Chen serves as Professor of Pediatrics at UCLA's David Geffen School of Medicine and Chief of Pediatric Neurology at Harbor-UCLA Medical Center. As an Investigator at The Lundquist Institute, she leads research in rare neurogenetic diseases through dual appointments in the Translational Genomics and Neurotherapeutics Institutes.

She earned her MD from Boston University School of Medicine and completed integrated residency training in Pediatrics and Adult/Child Neurology at Harbor-UCLA Medical Center, joining the faculty immediately afterward.

Her research targets central nervous system manifestations in lysosomal storage disorders with focus on:

• Mucopolysaccharidosis types I and III (Sanfilippo syndrome)
• Niemann-Pick disease type C
• Intrathecal delivery systems for enzyme replacement
• Neuroinflammation modulation using repurposed therapeutics
• Biomarker development for treatment monitoring

Dr. Chen maintains active grant funding evidenced by ongoing clinical trials including the 2024 Anakinra phase 1/2 study. Her publication record demonstrates consistent high-impact output in journals like Nature Medicine and The Lancet.

She conducts translational research within The Lundquist Institute's collaborative ecosystem, leveraging institutional resources for rare disease therapeutics development while mentoring the next generation of pediatric neurologists.