Drugs / TYF-ARSA
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| — | NCT07046338 | Jun 2025 overdue | metachromatic leukodystrophy | Shenzhen Geno-Immune Medical Institute | Recruiting | No outcome recorded |
| — | NCT03725670 | May 2025 → Dec 2029 expected | metachromatic leukodystrophy | Shenzhen Geno-Immune Medical Institute | 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 | TYF-ARSA | “safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect” NCT03725670 ↗ |
| Action | Restore | “to functionally correct the genetic defect” NCT03725670 ↗ |
| Route | Intrathecal | “by intrathecal (IT) and intravenous (IV) injections” NCT03725670 ↗ |
| Route | Intravenous | “by intrathecal (IT) and intravenous (IV) injections” NCT03725670 ↗ |
| Target | ARSA | “lentiviral vector (LV) carrying a functional ARSA gene” NCT07046338 ↗ |