Drug Type AAV based gene therapy |
Synonyms- |
Target |
Action modulators |
Mechanism SANS modulators(USH1 protein network component sans modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Leukodystrophy, Metachromatic | Preclinical | France | 28 Feb 2023 |





