Drug Type ASO |
Synonyms single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene (SynaptixBio) |
Target |
Mechanism TUBB4A inhibitors(tubulin beta 4A class IVa inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Leukodystrophy, Hypomyelinating, 6 | Preclinical | GB | 14 Oct 2021 |