Drugs / ASO-GNAO1
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07363603 | Sep 2025 → Dec 2026 expected | developmental and epileptic encephalopathy, 17, neurodevelopmental disorder with hyperkinetic movements and dyskinesia | Pirogov Russian National Research Medical University | Recruiting | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ASO-GNAO1 | “ASO-GNAO1 (Tianasen) is an investigational antisense oligonucleotide (ASO) therapy designed for the treatment of GNAO1-encephalopathy.” NCT07363603 ↗ |
| Action | Inhibit | “allele-specific suppression of the mutant GNAO1 protein expression” NCT07363603 ↗ |
| Modality | Antisense oligonucleotide | “an investigational antisense oligonucleotide (ASO) therapy” NCT07363603 ↗ |
| Route | Intrathecal | “intrathecal administration of ASO-GNAO1” NCT07363603 ↗ |
| Target | GNAO1 | “suppression of the mutant GNAO1 protein expression” NCT07363603 ↗ |