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GH001 is an investigational drug candidate consisting of mebufotenin (5-methoxy-N,N-dimethyltryptamine or 5-MeO-DMT), a potent naturally occurring psychedelic compound. Developed by GH Research, it is formulated for administration via a proprietary pulmonary inhalation approach. The drug is designed to provide an ultra-rapid antidepressant effect through an individualized dosing regimen (IDR) that allows for the administration of up to three escalating doses (6 mg, 12 mg, and 18 mg) within a single day. GH001 primarily targets the serotonin system, acting as an agonist at 5-HT2A and 5-HT1A receptors, which is believed to mediate its rapid therapeutic effects in psychiatric disorders. It is currently being evaluated in clinical trials for treatment-resistant depression, bipolar II disorder with current major depressive episodes, and postpartum depression.
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