Drug Type AAV based gene therapy  | 
Synonyms-  | 
Target  | 
Action modulators  | 
Mechanism C9orf72 modulators(C9orf72-SMCR8 complex subunit modulators)  | 
Active Indication  | 
Inactive Indication-  | 
Originator Organization  | 
Active Organization  | 
Inactive Organization-  | 
License Organization-  | 
Drug Highest PhasePreclinical  | 
First Approval Date-  | 
Regulation-  | 
| Indication | Highest Phase | Country/Location | Organization | Date | 
|---|---|---|---|---|
| Amyotrophic Lateral Sclerosis | Preclinical | United Kingdom   | 22 Sep 2024 | 





