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VQB-5812
- Development stage
- Unknown
- Lead developer
- Vanqua Bio
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
VQB-5812 is a brain-penetrant small molecule activator of the enzyme glucosylceramidase (GCase), developed by Vanqua Bio for the treatment of Parkinson's disease associated with mutations in the GBA1 gene (GBA-PD). Mutations in GBA1 are the most common genetic risk factor for Parkinson's, leading to reduced GCase activity, lysosomal dysfunction, and the toxic accumulation of alpha-synuclein. VQB-5812 is designed to restore or enhance GCase enzymatic activity, thereby promoting the clearance of glycosphingolipids and preventing neurodegeneration. The compound is currently in Phase 1 clinical development to assess its safety, tolerability, and pharmacological profile in humans.
02
Targets
GBA1 (Glucosylceramidase)
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