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Lysosomal Diseases

Gordon Research Conference

Biology, Pathophysiology and Therapeutics of Lysosome-Associated Diseases

March 2 - 7, 2025

Chairs

and

Vice Chairs

and

Sheraton Fairplex Hotel & Conference Center

601 West McKinley Ave.

Pomona, California, United States

Venue and Travel Information

Conference Description

The Lysosomal Diseases GRC is a premier, international scientific conference focused on advancing the frontiers of science through the presentation of cutting-edge and unpublished research, prioritizing time for discussion after each talk and fostering informal interactions among scientists of all career stages. The conference program includes an array of speakers and discussion leaders from institutions and organizations worldwide, concentrating on the latest developments in the field. The conference is five days long and held in a remote location to increase the sense of camaraderie and create scientific communities, with lasting collaborations and friendships. In addition to premier talks, the conference has designated time for poster sessions from individuals of all career stages, and afternoon free time and communal meals allow for informal networking opportunities with leaders in the field.

This meeting is the premier independent international forum for discussion of cutting-edge research on the biology of lysosomes and and its clinical application: the concept is to link function to pathophysiology for innovative therapies. The program will cover the latest discoveries that advance knowledge about basic lysosomal function; thoroughly explore how function is impaired in lysosomal diseases - and how this science can be exploited to inform more universal application in biology and medicine. Whenever possible, advances in science will be linked to deep understanding of the clinical phenotype and authentic therapeutic discovery. The widest participation from molecular cell biologists, geneticists, clinical investigators and biochemists is envisaged. The meeting will also shed light on pathological aspects that extend beyond storage - including the extended role of the lysosome in neurodegeneration, energy metabolism, infection and immunity.

Related Meeting

This GRC will be held in conjunction with the "Lysosomal Diseases" Gordon Research Seminar (GRS). Those interested in attending both meetings must submit an application for the GRS in addition to an application for the GRC. Refer to the associated GRS program page for more information.

Conference Program

Sunday
2:00 pm - 9:00 pm	Arrival and Check-in
6:00 pm - 7:00 pm	Dinner
7:30 pm - 7:40 pm	Introductory Comments by GRC Staff / Welcome and Introduction from the Chairs
7:40 pm - 9:30 pm	Keynote Session: Lysosomal Function in Health and Disease Discussion Leader:
7:40 pm - 7:45 pm	Introduction by Discussion Leader
7:45 pm - 8:10 pm	"Developing Gene Therapies to Treat Dysfunctional Brain Circuits"
8:10 pm - 8:20 pm	Discussion
8:20 pm - 8:45 pm	"Mechanisms and Consequences of latrogenic Lysosomal Storage"
8:45 pm - 8:55 pm	Discussion
8:55 pm - 9:20 pm	"Intra-Lysosomal Lipid Metabolism and Neurodegeneration"
9:20 pm - 9:30 pm	Discussion
Monday
7:30 am - 8:30 am	Breakfast
9:00 am - 12:30 pm	Lysosomal Protein Function and Disease Mechanisms Discussion Leader:
9:00 am - 9:05 am	Introduction by Discussion Leader
9:05 am - 9:30 am	"Role of Lysosomal Transporters for Protein Catabolites in Cellular and Organismal Nutrition"
9:30 am - 9:35 am	Discussion
9:35 am - 10:00 am	"Lysosome Dynamics in C. Elegans Development and Aging"
10:00 am - 10:05 am	Discussion
10:05 am - 10:30 am	"Structural Studies of the Lysosomal Multienzyme Complex"
10:30 am - 10:35 am	Discussion
10:35 am - 11:00 am	Coffee Break
11:00 am - 11:25 am	"Decoding Cholesterol-mTORC1 Signaling in Organelle Homeostasis and Neurodegeneration"
11:25 am - 11:30 am	Discussion
11:30 am - 11:45 am	"Mechanosensitive Ion Channel TMEM63 Modulate Lysosomal Morphology and Function"
11:45 am - 11:50 am	Discussion
11:50 am - 12:00 pm	"Investigating the Role of LncRNAs in Lysosome Health and Function"
12:00 pm - 12:05 pm	Discussion
12:05 pm - 12:30 pm	Poster Previews
12:30 pm - 1:30 pm	Lunch
1:30 pm - 4:00 pm	Free Time
4:00 pm - 6:00 pm	Poster Session
6:00 pm - 7:00 pm	Dinner
7:30 pm - 9:30 pm	Novel Aspects in Lysosome Biology Discussion Leader:
7:30 pm - 7:35 pm	Introduction by Discussion Leader
7:35 pm - 8:00 pm	"Interactomics Identifies a Novel Role for the PD/DLB-Linked VPS13C in Non-Canonical mTORC1 Signaling"
8:00 pm - 8:05 pm	Discussion
8:05 pm - 8:30 pm	"Decoding Lysosomal Mechanisms of Cellular Adaptation"
8:30 pm - 8:35 pm	Discussion
8:35 pm - 9:00 pm	"STING Mediates Lysosomal Quality Control and Recovery through its Proton Channel Function and TFEB Activation"
9:00 pm - 9:05 pm	Discussion
9:05 pm - 9:25 pm	"JIP4 Promotes Lysosomal Cystine Efflux by Suppressing TMEM55-Dependent Cystinosin (CTNS) Degradation"
9:25 pm - 9:30 pm	Discussion
Tuesday
7:30 am - 8:30 am	Breakfast
8:30 am - 9:00 am	Group Photo
9:00 am - 12:30 pm	Lysosomal Diseases and What We Learn from Preclinical Studies Discussion Leader:
9:00 am - 9:10 am	Introduction by Discussion Leader
9:10 am - 9:35 am	"Platform AAV-Based Gene Therapy Approach for Multiple Lysosomal Storage Diseases"
9:35 am - 9:40 am	Discussion
9:40 am - 10:05 am	"Project Butterfly: Making a Personalized Antisense Oligonucleotide Medicine for CLN3 Batten Disease"
10:05 am - 10:15 am	Discussion
10:15 am - 10:30 am	"Combining Stabilized Glucocerebrosidase with a Blood-Brain Barrier Transport Vehicle Enhances Brain Delivery of Active Enzyme in Preclinical Models of Gaucher and Parkinson’s Diseases"
10:30 am - 10:35 am	Discussion
10:35 am - 11:00 am	Coffee Break
11:00 am - 11:15 am	"Preclinical Studies Evaluating the Biodistribution, Safety, and Pharmacodynamics of LTS-101, a Novel Gene Therapy for CLN2 Batten Disease"
11:15 am - 11:20 am	Discussion
11:20 am - 11:35 am	"Developing a dual AAV gene therapy to treat galactosialidosis and sialidosis using small and large animal models."
11:35 am - 11:40 am	Discussion
11:40 am - 11:55 am	"Systematic analysis of cellular proteomes and lipidomes in lysosomal storage disease."
11:55 am - 12:00 pm	Discussion
12:00 pm - 12:10 pm	"Uncovering the Role of TFEB in Pathogenesis and Disease Progression in Tuberous Sclerosis using Novel Human Kidney Organoid Models"
12:10 pm - 12:15 pm	Discussion
12:15 pm - 12:25 pm	"Spatial and Functional Separation of mTORC Signaling in Response to Different Amino Acid Sources"
12:25 pm - 12:30 pm	Discussion
12:30 pm - 1:30 pm	Lunch
1:30 pm - 4:00 pm	Free Time
4:00 pm - 6:00 pm	Poster Session
6:00 pm - 7:00 pm	Dinner
7:30 pm - 9:30 pm	Lysosomal Processes and Biomarkers Implicated in the Pathogenesis of Rare and Common Disorders Discussion Leader:
7:30 pm - 7:40 pm	Introduction by Discussion Leader
7:40 pm - 8:05 pm	"AAV Gene Therapy for Tay-Sachs and Sandhoff Disease: From Mice to Man and a Few Large Animal Models In-Between"
8:05 pm - 8:10 pm	Discussion
8:10 pm - 8:30 pm	"Integrated Multi-Omics Analysis using MENTOR Reveals Metabolic Reprogramming in the Niemann-Pick Type C Mouse Brain"
8:30 pm - 8:35 pm	Discussion
8:35 pm - 8:55 pm	"Cell Autonomous Roles of Galactosylceramidase and Contribution to Krabbe Disease"
8:55 pm - 9:00 pm	Discussion
9:00 pm - 9:25 pm	"Delineating the Molecular Mechanism Driving Neuroinflammation and Neurodegeneration in CLN3 Disease Pathophysiology"
9:25 pm - 9:30 pm	Discussion
Wednesday
7:30 am - 8:30 am	Breakfast
9:00 am - 12:30 pm	Therapies for Lysosomal Diseases and Clinical Trials Discussion Leader:
9:00 am - 9:05 am	Introduction by Discussion Leader
9:05 am - 9:30 am	"Development of Lentiviral Gene Therapy for Pompe Disease and Hunter Syndrome for Transparent and Reasonable Pricing"
9:30 am - 9:35 am	Discussion
9:35 am - 9:55 am	"Technological and Regulatory Hurdles in the Development of Gene Therapy Drugs for Rare Disease"
9:55 am - 10:00 am	Discussion
10:00 am - 10:25 am	"Advances in Our Understanding and Treatment of Niemann-Pick Disease Type C"
10:25 am - 10:30 am	Discussion
10:30 am - 11:00 am	Coffee Break
11:00 am - 11:20 am	"A Newly Engineered AAV in Non-Human Primates Allows Robust and Therapeutically Relevant Gene Expression to Treat Late Infantile Neuronal Ceroid Lipofuscinosis"
11:20 am - 11:25 am	Discussion
11:25 am - 11:45 am	"Domain-Substituted IGF2 Tag to Modulate Receptor Targeting during Lentiviral Gene Therapy for Mucopolysaccharidosis Type II"
11:45 am - 11:50 am	Discussion
11:50 am - 12:05 pm	"Efficacy of AAV-Mediated Gene Therapy in a Sheep Model of CLN1 Disease"
12:05 pm - 12:10 pm	Discussion
12:10 pm - 12:25 pm	"In House Development of Gene Therapy for Pompe Disease: Preclinical Data and Lessons Learned"
12:25 pm - 12:30 pm	Discussion
12:30 pm - 1:30 pm	Lunch
1:30 pm - 4:00 pm	Free Time
4:00 pm - 6:00 pm	Poster Session
6:00 pm - 7:00 pm	Dinner
7:30 pm - 9:30 pm	Lysosomes in Neurodegeneration and Underdiagnosed Lysosomal Diseases Discussion Leader:
7:30 pm - 7:35 pm	Introduction by Discussion Leader
7:35 pm - 8:00 pm	"Heterozygosity of Lysosomal Enzyme Genes and Adult-Onset Neurodegenerative Disease"
8:00 pm - 8:05 pm	Discussion
8:05 pm - 8:30 pm	"Newly Discovered Glycosylated Metabolites Changing Paradigms of Gaucher Disease"
8:30 pm - 8:35 pm	Discussion
8:35 pm - 9:00 pm	"Key Lysosomal Proteins in Parkinson's Disease Interact to Regulate Polyamine Homeostasis"
9:00 pm - 9:05 pm	Discussion
9:05 pm - 9:25 pm	"Modulation of Neuronal Lysosome Positioning and Function Resolves Alzheimer’s Disease-Linked Pathologies in Cultured Human Neurons"
9:25 pm - 9:30 pm	Discussion
Thursday
7:30 am - 8:30 am	Breakfast
8:30 am - 9:00 am	Business Meeting Nominations for the Next Vice Chair(s); Complete the GRC Evaluation Forms; Election of the Next Vice Chair(s)
9:00 am - 12:30 pm	Gene Editing and Novel Therapies for Lysosomal Storage Diseases Discussion Leader:
9:00 am - 9:10 am	Introduction by Discussion Leader
9:10 am - 9:35 am	"Base Editing and Prime Editing to Correct Disease-Causing Genetic Variants"
9:35 am - 9:40 am	Discussion
9:40 am - 10:05 am	"Somatic Genome Editing as Therapy for Lysosomal Storage Disorders"
10:05 am - 10:10 am	Discussion
10:10 am - 10:30 am	"Novel Therapeutic Strategies for Niemann Pick Type C Disease"
10:30 am - 10:35 am	Discussion
10:35 am - 11:00 am	Coffee Break
11:00 am - 11:20 am	"Exploring the Pathophysiological Basis of Dysphagia in CLN3 Deficient Mice and the Therapeutic Potential of Gene Therapy"
11:20 am - 11:25 am	Discussion
11:25 am - 11:45 am	"ACTH4-7PGP, a Promising Small Molecule for Treatment of Sanfilippo Disease"
11:45 am - 11:50 am	Discussion
11:50 am - 12:05 pm	"Development of Novel TRPML1 Activators that Modulate Endolysosomal Pathways and Assessment in Disease Models"
12:05 pm - 12:10 pm	Discussion
12:10 pm - 12:25 pm	"Mechanism and rescue of lysosomal defects in Adult-onset Neuronal Ceroid Lipofuscinosis"
12:25 pm - 12:30 pm	Discussion
12:30 pm - 1:30 pm	Lunch
1:30 pm - 4:00 pm	Free Time
4:00 pm - 6:00 pm	Poster Session
6:00 pm - 7:00 pm	Dinner
7:30 pm - 9:30 pm	Late-Breaking Discoveries in Lysosomal Diseases Discussion Leader:
7:30 pm - 7:35 pm	Introduction by Discussion Leader
7:35 pm - 8:00 pm	"Subcellular Separation of Nutrient Signaling to mTORC1"
8:00 pm - 8:05 pm	Discussion
8:05 pm - 8:30 pm	"Novel Human Receptor-Targeted Capsids for CNS Gene Therapy"
8:30 pm - 8:35 pm	Discussion
8:35 pm - 8:55 pm	"Cell-Type Resolved Protein Atlas of Brain Lysosomes Identifies SLC45A1-Associated Disease as a Lysosomal Disorder"
8:55 pm - 9:00 pm	Discussion
9:00 pm - 9:20 pm	"Identifying and Targeting Novel Lysosomal Enzymes in Pancreatic Cancer"
9:20 pm - 9:25 pm	Discussion
9:25 pm - 9:30 pm	Closing Remarks
Friday
7:30 am - 8:30 am	Breakfast
9:00 am	Departure

Contributors

Phoenix Nest Inc.

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Conference History Lysosomal Diseases (GRS)

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