Drugs / RB001
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT07690527 | Aug 2026 → Jul 2031 expected | Phelan-McDermid syndrome due to SHANK3 mutation | Peking University First Hospital | Enrolling by invitation | No outcome recorded |
| Phase 1 | NCT07014020 | Jun 2025 → May 2027 expected | Phelan-McDermid syndrome due to SHANK3 mutation | Peking University First Hospital | Active not recruiting | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RB001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07014020 ↗ |
| Action | Restore | “a single ICV injection of RB001 could restore SHANK3 mRNA and protein expression” NCT07014020 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “RB001 utilizes the Adeno-Associated Virus (AAV) vector to deliver an optimized SHANK3-minigene” NCT07014020 ↗ |
| Route | Other | “administered via intracerebroventricular (ICV) injection” NCT07014020 ↗ |
| Target | SHANK3 | “RB001 utilizes the Adeno-Associated Virus (AAV) vector to deliver an optimized SHANK3-minigene” NCT07014020 ↗ |