| Drug Type Small molecule drug | 
| Synonyms- | 
| Target | 
| Action modulators | 
| Mechanism C9orf72 gene 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 | Italy  | 07 May 2025 | |
| Frontotemporal Dementia | Preclinical | Italy  | 07 May 2025 | 





