7:30
9:00
Registration & Breakfast
9:50 – 10:10
10:10 – 10:30
Lessons from the ACTIVATE trial
Overview of ongoing trials and preclinical effortson the ACTIVATE trial
11:00 – 11:20
11:20 – 11:40
11:40 – 12:00
12:00 – 12:20
12:20 – 12:30
Update from Company 1
Update from Company 2
Update from Company 3
Update from Company 4
Q&A
12:30
14:30
Lunch + Poster session
14:30 – 14:40
14:40 – 14:50
14:50 – 15:00
15:00 – 15:10
15:10 – 15:20
15:20 – 15:30
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
16:00 – 16:20
16:20 – 16:40
16:40 – 17:00
Patient perspective on GBA1 research
Patient advocacy group perspective
Research foundation perspective
18:00
21:00
Cocktail Reception
08:00
09:00
Registration & Breakfast
9:10 – 9:30
9:30 – 9:50
9:50-10:00
Novel insights into the natural history of GBA1-PDpresentation – Base editing for GD and GBA1-associated PD: lessons from Tay Sachs
Emerging biomarkers for GBA1-PD
Q&A
10:30 – 10:40
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
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
Selected from abstracts
12:00
14:00
Lunch + Poster session
14:00 – 14:20
14:20 – 14:40
14:40 – 15:00
15:00 – 15:20
15:20 – 15:30
Update from Company 5
Update from Company 6
Update from Company 7
Update from Company 8
Q&A
-
16:00 – 16:20
16:20 – 16:40
16:40 – 17:00
Brain isoform of GBA1 – what we know and what we don’t know
Are GBA1 isoforms relevant for GBA1-PD?
Q&A
-
8:00
9:00
Registration & Breakfast
9:00 – 9:20
9:20 – 9:40
9:40 – 10:00
GBA1, lipids and metabolites 1
GBA1, lipids and metabolites 2
GBA1, lipids and metabolites 3
10:30 – 10:40
10:40 – 10:50
10:50 – 11:00
11:00 – 11:10
11:10 – 11:20
11:20 – 11:30
Selected from projects funded by the 2026 GBA1 call
Selected from projects funded by the 2026 GBA1 call
Selected from projects funded by the 2026 GBA1 call
Selected from projects funded by the 2026 GBA1 call
Selected from projects funded by the 2026 GBA1 call
Selected from projects funded by the 2026 GBA1 call
11:30 – 11:45
11:45 – 12:00
12:00 – 12:15
12:15 – 12:30
Ambroxol – mechanism of action in GBA1-PD
Overview of results from ambroxol trials – lessons so far
Ongoing trials and GBA1 mutation selection
Discussion
12:30
14:00
Lunch + Poster session
14:00 – 14:15
14:15 – 14:30
14:30 – 14:45
14:45 – 15:00
How does TMEM175 modify GBA1 activity
Mutation spectrum and clinical correlations
Novel mechanistic insights on TMEM175 - 1
Novel mechanistic insights on TMEM175 - 2
15:30 – 15:45
15:45 – 16:00
16:00 – 16:15
16:15 – 16:30
16:30 – 16:40
Brain delivery – viral capsids and LNPs
Is gene editing a viable therapy option?
Cell therapy for GBA1-PD
RNA therapy - modifying the modifiers
Q&A
16:40
17:00
Concluding remarks