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alogabat
- Development stage
- Phase 2
- Lead developer
- Roche
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Alogabat is an investigational small molecule drug that acts as a positive allosteric modulator of the α5 subunit-containing GABAA receptor. It is being developed primarily for the treatment of Angelman syndrome, particularly in children and adolescents with the deletion genotype, and is also under investigation for pervasive developmental disorders such as autism. By enhancing the function of remaining α5-containing GABAA receptors—which are reduced in individuals with certain genetic deletions—alogabat aims to improve neurological symptoms associated with these conditions. The drug is administered orally and is currently in phase 2 clinical trials led by Roche[1][2][4][5][6].
- Other names
- GABA-Aa5 PAMGABA-Aa-5 PAMGABA-Aa 5 PAMGABA-Aα5 PAM
02
Targets
GABRA5 (Gamma-aminobutyric acid type A receptor subunit alpha-5)
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