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 diversity and inclusion in the scientific workplace by providing a safe environment for informal and meaningful conversations amongst colleagues of all career stages. The program supports the professional growth of all members of our communities, including ethnicity, race and/or gender identity by providing an open forum for discussion and mentoring.
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