Satellite meeting of World Parkinson’s Congress
7:30
9:00
Registration & Breakfast
Recent developments in GBA1 research and future perspectives
9:40 – 10:00
10:00 – 10:20
10:20 – 10:30
Overview of GBA1-targeting trialsGBA1-targeting trials
Report on the ACTIVATE trial
Q&A
Simon Stott, Cure Parkinson's
Tiago Fonseca, Bial
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11:00 – 11:20
11:20 – 11:40
11:40 – 12:00
12:00 – 12:20
12:20 – 12:30
The cholinergic system in GBA1-PD
GBA1 and Lysosomal Oligogenic Risk for Parkinson’s Disease
Targeting Lysosomal pH Restores Mitochondrial Quality Control in GBA1-Mutant Parkinson’s Disease
Lysosomal glucocerebrosidase is needed for ciliary Hedgehog signaling
Q&A
Teus van Laar, University of Groningen
Joshua Shulman, Baylor College of Medicine
Preethi Sheshadri, UK Dementia Research Institute Sheshadri, UK Dementia Research Institute
Suzanne Pfeffer, Stanford University
12:30
12:40
Lunch + Poster session (Posters 2201-2215)
14:30 – 14:40
14:40 – 14:50
14:50 – 15:00
15:00 – 15:10
15:10 – 15:20
15:20 – 15:30
Liver-direct gene therapy enables brain delivery of GCase and extends survival in neuronopathic Gaucher disease mice
Longitudinal IQ and olfactory assessments in GBA1 variant carriers with and without Parkinson disease
Functional evaluation of Parkinson’s disease-associated SMPD1 variants in disease-relevant cell types
Development of a small molecule TRPML1 agonist for Parkinson’s disease
SIM0817: Small Molecule GCase Activator for the Treatment of Parkinson’s Disease: Improved Potency and Favorable Druggability
Genetic screening in people with Parkinson’s disease: challenges and solutions in identification of gba1 mutation carriers for future clinical trials
Maria Laura Allende, National Institutes of Health
Ellen Sidransky, National Institutes of Health
Elaine Guo Yan Chew, Lee Kong Chian School of Medicine, Singapore
Valerie Cullen, Lysoway Therapeutics Inc
Xiaoyuan Wei, Simcere Pharmaceutical
Jurrian van der Valk, Centre for Human Drug Research
16:00 – 16:15
16:15 – 16:30
16:30 – 16:45
16:45 – 17:00
Venglustat in GD – implications for GD and PD
An update on the development of the GCase modulator GT-02287 for Parkinson’s disease
Vq-101, an allosteric activator of lysosomal glucocerebrosidase, shows sustained target and pathway engagement in patients with Parkinson’s disease
Q&A
Pramond Mistry, Yale School of Medicine
Jonas Hannestad, Gain Therapeutics
Daniel Ysselstein, Vanqua Bio
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18:00
21:00
Cocktail Reception + meet the experts
08:00
09:00
Registration & Breakfast
9:10 – 9:40
9:40 – 10:00
10:00 – 10:10
Keynote presentation – Base editing for GD and GBA1-associated PD: lessons from Tay Sachs
GBA1 in neurons vs astrocytes
Q&A
Richard Proia, National Institutes of Health (NIH)
Marie Davis, University of Washington
10:40 – 10:50
10:50 – 11:00
11:00 – 11:10
11:10 – 11:20
11:20 – 11:30
11:30 – 11:40
11:40 – 11:50
11:50 – 12:00
Development of an efficacious systemic AAV gene therapy for neuronopathic Gaucher disease with applicability to GBA1-associated PD
GBA2 as a regulator of glycosphingolipid synthesis and Gaucher disease pathogenesis
Glycosphingolipid dysregulation in GBA1-PD: combined plasma and brain analysis
Neurotinib, a brain penetrant c-abl inhibitor, prevents activation and phosphorylation of c-abl kinase and a-synuclein, in in vivo and in vitro models
A Novel Regulator of GCase: CLCN3 Regulates Lysosomal Function in Parkinson’s Disease
Single-cell RNA sequencing analysis of the peripheral immune system in idiopathic, LRRK2 and GBA1 Parkinson’s disease
Interim Results from the AGP1 Trial: High-Dosage-Ambroxol in GBA1-Associated Parkinson’s Disease (Sidransky syndrome)
Linking PD GENEration Genetic Testing with Continuous Digital Phenotyping to Characterize GBA1 Variant-Associated Progression in Parkinson Disease
Ellen Sidransky, National Institutes of Health
Hannah Duffy, National Institutes of Health
Linda Montavoci, Oxford University
Nahid Tayebi, National Institutes of Health
Donovan Worrall, McGill University
Mikaela Rosen Perez, Icahn School of Medicine
Ari Zimran, Shaare Zedek Medical Center
Ro'ee Gilron, Rune Labs
12:00
14:00
Lunch + Poster session (Posters 2301-2316)
14:00 – 14:20
14:20 – 14:40
14:40 – 15:00
15:00 – 15:20
15:20 – 15:30
The global burden of GBA1-PD – clinical implications
Treatment of non-motor symptoms in GBA1-PD
Biomarkers for GBA1-PD
Challenges in genetic counseling for GBA1 in PD
Q&A
Ziv Gan-Or, McGill University
Avner Thaler, Tel Aviv University
Fernanda Cerqueira, The Michael J. Fox Foundation
Valentina Caceres, Indiana University School of Medicine
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16:00 – 16:20
16:20 – 16:40
16:40 – 16:50
16:50 – 17:00
Bidirectional regulation of GPNMB by β- glucocerebrosidase deficiency
CRISPR screening for modifiers of GCase activity
Q&A
Concluding remarks
Yu Chen, National Institutes of Health (NIH)
Melissa Roberts, Denali Therapeutics
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Ziv Gan-Or, McGill University
18:00
20:00
Goodbye cocktail