Drug Type AAV based gene therapy |
Synonyms RM-101 |
Target |
Mechanism HBG gene stimulators, usherin modulators(Usherin modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseIND Approval |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Usher Syndrome type 2 | IND Approval | CN | 12 Oct 2024 | |
Usher Syndromes | IND Approval | US | 17 Sep 2024 |