Drugs / RO7248824
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT04863794 | Apr 2021 → Aug 2022 | Angelman syndrome | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT04428281 | Aug 2020 → Jul 2025 | Angelman syndrome | Hoffmann-La Roche | Completed | Met primary |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RO7248824 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04428281 ↗ |
| Known as | rugonersen | “Rugonersen (RO7248824) is an antisense oligonucleotide that reinstates UBE3A by derepressing the silenced paternal allele.” PMID 40646322 ↗ Jul 2025 |
| Action | Restore | “reinstates UBE3A by derepressing the silenced paternal allele” PMID 40646322 ↗ Jul 2025 |
| Route | Intrathecal | “RO7248824 administered intrathecally (IT)” NCT04428281 ↗ |