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
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
9:00 am - 9:25 am
"Role of Lysosomal Transporters for Protein Catabolites in Cellular and Organismal Nutrition"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
"Lysosome Dynamics in C. Elegans Development and Aging"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
"Structural Studies of the Lysosomal Multienzyme Complex"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Late-Breaking Topic
11:25 am - 11:30 am
Discussion
11:30 am - 11:45 am
Short Talk Selected from Poster Abstracts
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
Short Talk Selected from Poster Abstracts
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
Short Talk Selected from Poster Abstracts
12:25 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hourâ„¢
The GRC Power Hourâ„¢ is designed to address the interests and needs of all scientists and the challenges they face in their careers. Specifically, the program promotes meaningful conversations amongst colleagues of all career stages about barriers to career advancement and strategies to support the professional growth of all members of the community. The GRC Power Hourâ„¢ is open to all conference participants.
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
7:30 pm - 7:55 pm
"Interactomics Identifies a Novel Role for the PD/DLB-Linked VPS13C in Non-Canonical mTORC1 Signaling"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
"Decoding Lysosomal Mechanisms of Cellular Adaptation"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
"BORC-Deficiency Syndrome: A Novel Neurodevelopmental Disorder Caused by Defective Lysosome Dynamics"
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:25 pm
Late-Breaking Topic
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
9:00 am - 9:25 am
"Platform AAV-Based Gene Therapy Approach for Multiple Lysosomal Storage Diseases"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
"Project Butterfly: Making a Personalized Antisense Oligonucleotide Medicine for CLN3 Batten Disease"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
Late-Breaking Topic
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Late-Breaking Topic
11:25 am - 11:30 am
Discussion
11:30 am - 11:45 am
Short Talk Selected from Poster Abstracts
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
Short Talk Selected from Poster Abstracts
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
Short Talk Selected from Poster Abstracts
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
7:30 pm - 7:55 pm
"Biomarker Discovery and Concurrent Phenotyping in CLN3 Disease"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
"Delineating the Molecular Mechanisms Driving Neuroinflammation and Neurodegeneration in CLN3 Disease Pathophysiology"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Late-Breaking Topic
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:25 pm
Late-Breaking Topic
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
9:00 am - 9:25 am
"AAV Gene Therapy for Tay-Sachs and Sandhoff Disease: From Mice to Man and a Few Large Animal Models In-Between"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
"Clinical Trials in Mucopolysaccharidoses"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
"Development of Lentiviral Gene Therapy for Pompe Disease and Hunter Syndrome for Transparent and Reasonable Pricing"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Late-Breaking Topic
11:25 am - 11:30 am
Discussion
11:30 am - 11:45 am
Short Talk Selected from Poster Abstracts
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
Short Talk Selected from Poster Abstracts
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
Short Talk Selected from Poster Abstracts
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
7:30 pm - 7:55 pm
"Heterozygosity of Lysosomal Enzyme Genes and Adult-Onset Neurodegenerative Disease"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
"Gaucher Disease: How a Rare Disease Provides a Window into Common Neurodegenerative Disorders"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Late-Breaking Topic
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:25 pm
Late-Breaking Topic
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
9:00 am - 9:25 am
"Somatic Genome Editing as Therapy for Lysosomal Storage Disorders"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
"CNS-Targeted Base Editing of the Major Late-Onset Tay-Sachs Mutation"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
"Base Editing and Prime Editing to Correct Disease-Causing Genetic Variants"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Late-Breaking Topic
11:25 am - 11:30 am
Discussion
11:30 am - 11:45 am
Short Talk Selected from Poster Abstracts
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
Short Talk Selected from Poster Abstracts
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
Short Talk Selected from Poster Abstracts
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
7:30 pm - 7:55 pm
"Subcellular Separation of Nutrient Signaling to mTORC1"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
"Novel Human Receptor-Targeted Capsids for CNS Gene Therapy"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Late-Breaking Topic
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:25 pm
Late-Breaking Topic
9:25 pm - 9:30 pm
Discussion
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure