GBA Therapeutics
This page collects therapeutic strategies from the GBA-PD pilot range. It is a source-cited map, not a final interpretation.
Strategy Map
| strategy | corpus representation | sources |
|---|---|---|
| Isofagomine | Appears in the summary area before eliglustat. | 20240722_181748 |
| Eliglustat | Covered through GD type 1 trial material and continuation into the next photo. | Eliglustat |
| Ambroxol | Covered by mechanism, preclinical evidence, and clinical-trial sections. | Ambroxol |
| Venglustat / ibiglustat | Covered by preclinical, in-house, biomarker, MOVES-PD, and clinical-trial sections. | Venglustat |
| PR001 / LY3884961 | Covered by preclinical mouse models, NHP ICM biodistribution, GCase activity/expression, lipid accumulation, insoluble alpha-synuclein, dose selection, and clinical-trial entry. | PR001 |
Pilot Comparison
| program | primary therapeutic idea in pilot | pilot caveat |
|---|---|---|
| Eliglustat | GCS inhibition in GD1, with marketed oral GD1 context and trials such as ENCORE, ENGAGE, EDGE, and earlier P2/P1 work. | Pilot pages are GD-heavy; PD relevance is marked uncertain or ? in the source summary. |
| Ambroxol | Pharmacological chaperone / GBA activator logic, including mutant-GCase folding and lysosomal delivery. | Source notes explicitly state null mutations may not benefit. |
| Venglustat | GCS inhibition; lipid lowering in GD/GBA-PD contexts and MOVES-PD. | MOVES-PD clinical outcome did not separate from placebo on MDS-UPDRS II+III in the cited 52-week result. |
| PR001 | AAV/GBA gene therapy aiming to raise GCase and influence downstream lipids/alpha-synuclein. | Source pages contain multiple dose bases and several high-risk vector-genome numeric values that need careful preservation. |
PR001 Notes
The seed PR001 evidence emphasizes whether gene therapy changes GCase activity/expression and downstream disease-relevant readouts. The corpus includes mouse genetic model data, A53T + CBE model material, NHP biodistribution, and dose-selection text. Sources: 20240722_181805, 20240722_181809.
Next Entity Pages
entities/compounds/isofagomine.mdafter more source evidence appears.