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AL0804
- Development stage
- Preclinical
- Lead developer
- AceLink Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
AL0804 is a highly brain-penetrant glucosylceramide synthase (GCS) inhibitor in development for the treatment of neuronopathic glycolipid storage diseases. Its primary indications include neuronopathic Gaucher disease (Type II and III), GM2 gangliosidosis (Tay-Sachs and Sandhoff disease), GM1 gangliosidosis, and certain genetic forms of Parkinson’s disease. The drug is designed to cross the blood-brain barrier effectively, targeting central nervous system manifestations of these disorders by inhibiting GCS, an enzyme involved in glycosphingolipid synthesis[1][9]. AL0804 is being developed by AceLink Therapeutics.
02
Targets
UGCG (UDP-glucose ceramide glucosyltransferase)
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