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
"Unanticipated Links Between Disparate Diseases: Insights From Lysosomal Storage Disorders"
8:10 pm - 8:20 pm
Discussion
8:35 pm - 9:00 pm
"The Lysosomal Multienzyme Complex: A Balancing Act Between Health and Disease"
9:00 pm - 9:10 pm
Discussion
9:10 pm - 9:35 pm
"Gaucher Disease: Glucosylceramide Accumulation and More"
9:35 pm - 9:45 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 - 9:20 am
"Insights into Lysosomal pH Disease from New Patients with Novel ClC-7 Gain-of-Function Mutations"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"LRRK2: A Brake For When Lysosomes Break"
9:50 am - 10:00 am
Discussion
10:00 am - 10:30 am
Coffee Break
10:30 am - 10:50 am
"Dissecting Lysosomal Cholesterol Signaling in Niemann-Pick Type C"
10:50 am - 11:00 am
Discussion
11:00 am - 11:20 am
"GBA1 Has a Functional Pseudogene Which Limits Our Understanding of Both its Function and Role in Disease"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
"Dysfunction of Lysosomal Proteins - Beyond Storage"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:20 pm
"Selective Modulation of mTORC1 Signaling at the Lysosome"
12:20 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hour™
The GRC Power Hour™ 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.
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Lysosomal Biogenesis and Trafficking
6:00 pm - 6:20 pm
"Learning from Yeast: Structure and Functions of HOPS in Membrane Fusion"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
"The HOPS Complex: Integrating Lysosomal Trafficking Pathways"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:20 pm
"LYSET - A Key Protein in the Biogenesis of Lysosomes and Viral Infection"
7:20 pm - 7:30 pm
Discussion
7:30 pm - 7:50 pm
"The Batten Disease Protein CLN3 Mediates Lysosomal Biogenesis and Reformation"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Tuesday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Group Photo
9:00 am - 12:30 pm
Lysosomal Processes Implicated in Pathogenesis
9:00 am - 9:30 am
"From Lysosomal Profiling to Novel Putative Lysosomal Diseases"
9:30 am - 9:40 am
Discussion
9:40 am - 10:00 am
"Lysosomes in TB Pathogenesis: The Bad, The Ugly and The Good"
10:00 am - 10:10 am
Discussion
10:10 am - 10:25 am
"Alterations in Lipid Homeostasis Affect Ragulator-Mediated Lysosomal Positioning"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
"Lysosomes Signal to Longevity Regulation"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
"Nutrient Regulated Control of Lysosome Function by Signaling Lipid Conversion"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:20 pm
"Intestinal Autophagic Defects, Microbiome Dysbiosis and Immune Activation in a Drosophila Model of Neuronopathic Gaucher Disease"
12:20 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Function of Lysosomal Enzymes In The Immune System
6:00 pm - 6:20 pm
"RNaseT2 Deficient Leukoencephalopathy – New Insights Including Type I Interferon Signaling"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
"Innate Immune Sensing of Lysosomal Dysfunction"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:20 pm
"Autoimmunity in Gaucher Disease"
7:20 pm - 7:30 pm
Discussion
7:30 pm - 7:50 pm
"ADA2 is a Lysosomal DNA-Binding Protein That Downregulates the Type 1 Interferon Response: Implications for Understanding and Therapy of the Autoinflammatory Disorder DADA2"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Small Molecule Therapies for Lysosomal Diseases
9:00 am - 9:20 am
"Small Molecules Therapy For Gaucher and Fabry Disease"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"Small Molecule Therapies for Pompe Disease"
9:50 am - 10:00 am
Discussion
10:00 am - 10:20 am
"Synthetic Analogue of Adrenocorticotropic Hormone Peptide Rescues Neurological Manifestations in Diseases of Mucopolysaccharidosis III Spectrum"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
"Small Molecules in the Treatment of Multiple Sulfatase Deficiency"
11:20 am - 11:30 am
Discussion
11:30 am - 11:45 am
"Towards a Substrate Reduction Therapy for Metachromatic Leukodystrophy Using a Small Molecule Inhibitor of UGT8"
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
"Structurally Targeted Allosteric Regulators Targeting GBA1 Diseases"
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
"AZ-3102- A Potent, Brain-Penetrant Inhibitor of Both GCS and GbA2 in Preclinical Mouse Models of Niemann-Pick Disease Type C and Sandhoff Disease"
12:25 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
6:00 pm - 6:20 pm
"AMPK Phosphorylation of FNIP1 Alters GAP Activity of the FLCN/FNIP1 Complex to Control Lysosome Biogenesis"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:40 pm
"Neu1 Modulates Trem2 Signaling and Microglial Function"
6:40 pm - 6:45 pm
Discussion
6:45 pm - 6:55 pm
"An SPNS1-Dependent Lysosomal Lipid Transport Pathway that Enables Cell Survival under Choline Limitation"
6:55 pm - 7:00 pm
Discussion
7:00 pm - 7:10 pm
"The Cholesterol Transporter NPC1 is Essential for Epigenetic Regulation and Maturation of Oligodendrocyte Lineage Cells"
7:10 pm - 7:15 pm
Discussion
7:15 pm - 7:25 pm
"Cell Autonomous Function of Galactosylceramidase in Krabbe Disease"
7:25 pm - 7:30 pm
Discussion
7:30 pm - 7:40 pm
"Pharmacological Chaperone Therapy for Aspartylglucosaminuria: Results of a Clinical Study with Cystadane/Betaine"
7:40 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
"Neurodegenerative Lysosomal Storage Disorders Damage the Enteric Nervous System, Impair Bowel Motility, and are Effectively Treated by Gene Therapy"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
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; Discuss Future Dates and Venue; Election of the Next Vice Chair(s)
9:00 am - 12:30 pm
Gene Therapy of Lysosomal Diseases
9:00 am - 9:20 am
"Lentiviral Stem Cell Gene Therapies for Neuronopathic Lysosomal Diseases"
9:20 am - 9:30 am
Discussion
9:30 am - 9:50 am
"Gene Therapy for Neuropathic LSDs"
9:50 am - 10:00 am
Discussion
10:00 am - 10:20 am
"Hematopoietic Stem Cell Gene Therapy for Cystinosis: Mechanism of Action, Clinical Translation, and Application to other Lysosomal Diseases"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
"Pompe Disease - What is in Store for the Next Decade?"
11:20 am - 11:30 am
Discussion
11:30 am - 11:45 am
"Applications for Gene Therapy for Lysosomal Disorders: From Capsid Selection to Efficacy Readouts"
11:45 am - 11:50 am
Discussion
11:50 am - 12:05 pm
"Transgene Expression Analysis to Support Therapeutic GBA1 Transfer Using LC-MS/MS"
12:05 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
"Development of a Novel Liver-Directed AAV Gene Therapy Candidate for Gaucher Disease Type I"
12:25 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Engineered Enzymes for Treatment of Lysosomal Diseases
6:00 pm - 6:20 pm
"Interim Biomarker and Clinical Data From a Phase 1/2 Study of Intravenous DNL310 (Brain-Penetrant Enzyme Replacement Therapy) in Mucopolysaccharidosis Type II"
6:20 pm - 6:30 pm
Discussion
6:40 pm - 7:00 pm
"Long-term Clinical Experience with Pabinafusp Alfa, an Approved CNS-Penetrating Enzyme"
7:00 pm - 7:10 pm
Discussion
7:10 pm - 7:30 pm
"Halting Fabry Disease Progression in a Symptomatic Disease Model With a rAAV Based Gene Therapy Expressing an Engineered Alpha Galactosidase A Protein"
7:30 pm - 7:40 pm
Discussion
7:40 pm - 7:55 pm
"Genetically Engineered Arylsulfatase A with Increased Activity, Stability and Transendothelial Transfer Rate for Treatment of Metachromatic Leukodystrophy"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:10 pm
Closing Remarks
8:00 pm - 9:00 pm
Dinner
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure