3rd Annual Robert J. Gorlin Symposium
Beyond the Blood Brain Barrier:
Strategies for Treating the CNS
Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
February 6, 2024
5:15 – 7:30 PM
The Robert J. Gorlin Symposium honors the work of Robert James Gorlin, DDS, PhD. Dr. Gorlin was a geneticist, maxillofacial pathologist, and academician at the University of Minnesota School of Dentistry. His groundbreaking research in genetic disorders of the head and neck, spanning over 50 years, revolutionized the understanding of the morphology of lysosomal diseases and many other genetic disorders. The 2nd Annual Robert J. Gorlin Symposium will focus on precision medicine in lysosomal diseases.
Overview
Each year, WORLDSymposium identifies an unmet need, or critical topic for presentation and discussion in the Annual Robert J. Gorlin Symposium. For 2024, this 2.25-hour non-CE session discussed current approaches for treating the central nervous system in lysosomal diseases. Presentations included an overview of current and future approaches for treating the central nervous system, and address challenges in identifying and implementing meaningful outcome measures for evaluating efficacy of central nervous system therapies.
The short-term mission of this session was to provide a broad, sweeping overview (“10,000-foot view”, or possibly even 30,000-foot view) of the current landscape for treating the central nervous system in lysosomal diseases, thereby giving a broad general impression of the current status of CNS treatments.
The long-term mission of this session was to set the stage for future, regularly-held meetings or workshops on this very topic. These future meetings will be designed for more in-depth coverage of key components, as well as generating white papers and creating a platform that will provide ongoing updates, and consensus-based levels of evidence.
Preliminary Agenda
Tuesday, February 6, 2024 (5:15 – 7:30 PM)
| 5:15 PM | Welcome (Jeanine R. Jarnes) |
| 5:17 PM | Introduction (Robert G. Thorne) |
| 5:20 PM | Overcoming challenges in quantifying developmental change for clinical trials (Elsa G. Shapiro) |
| 5:30 PM | Crossing the blood-brain barrier with in utero enzyme replacement therapy for lysosomal diseases (Tippi MacKenzie) |
| 5:40 PM | Intracerebroventricular infusion strategy for the delivery of cerliponase alfa to the central nervous system (Guillermo Seratti) |
| 5:50 PM | Venglustat and the Brain (Mario Aguiar) |
| 6:00 PM | Intra-cisterna magna administration of AAV gene therapy for neurodegenerative disorders (Samiah Al-Zaidy) |
| 6:10 PM | JCR Pharmaceuticals tailored Approaches for Treating the Central Nervous System Signs and Symptoms in neuronopathic Lysosomal Diseases (Mathias Schmidt) |
| 6:20 PM | Connecting the dots: Use of early biomarkers to predict longer term functional outcomes (Heather Lau) |
| 6:30 PM | Fabry in the CNS? (Biliana Veleva-Rotse) |
| 6:40 PM | Intracisternal administration of investigational AAV9 gene therapies to target the central nervous system in pediatric lysosomal disorders (Laura Pisani) |
| 6:50 PM | Physiologic determinants of treatment efficacy for neuropathic lysosomal storage disorders: Key considerations in going across or bypassing the blood-brain barrier (Robert G. Thorne) |
| 7:00 PM | Q & A |
| 7:30 PM | Adjourn |
Course Director:
Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Assistant Professor, Department of Pediatrics
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, United States
Co-Chairs:
Robert G. Thorne, PhD
Denali Fellow
Head, Denali Postdoc Program
Denali Therapeutics, South San Francisco, CA
Adjunct Associate Professor, Department of Pharmaceutics
University of Minnesota-Twin Cities, Minneapolis, MN
President, International Brain Barriers Society,
Editorial Board Member, Fluids & Barriers of the CNS
Elsa G. Shapiro, PhD, ABPP
Professor of Pediatrics and Neurology, Division of Pediatric Behavioral Neuroscience
University of Minnesota, Minneapolis, MN
Managing Partner at Shapiro Neuropsychology Consulting, LLC
Faculty
Tippi MacKenzie
Professor of Surgery
Division of Pediatric Surgery
UCSF School of Medicine
University of California, San Francisco
Guillermo Seratti
Medical Director
BioMarin Pharmaceutical Inc.
Mario Aguiar
Venglustat Medical Lead
Global Medical Affairs, Rare Diseases
Sanofi
Samiah Al-Zaidy
Vice President, Clinical Development
PassageBio
Mathias Schmidt
Vice President, Clinical Development, Global Business Strategy and Business Development
JCR Pharmaceuticals
Heather Lau
Executive Director, Global Clinical Development
Ultragenyx
Biliana Veleva-Rotse
Amicus Global Medical Director and Medicines Lead for Fabry
Amicus Therapeutics
Laura Pisani
Senior Medical Director, Clinical Development
REGENXBIO
Thank you to the sponsors for this session
