Last update 21 Nov 2024
TGT-001
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms a non-replicating recombinant adeno- associated virus serotype 9 (AAV9) based gene therapy vector containing the DNA of Streptococcus pyogenes Cas9 (SpCas9) protein and single guide RNA (sgRNA) designed to target the TATA-box of the PMP22 P1 promoter |
Target |
Mechanism PMP22 inhibitors(peripheral myelin protein 22 inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseIND Application |
First Approval Date- |
RegulationOrphan Drug (US) |
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R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Charcot-Marie-Tooth Disease, Type Ia | IND Application | KR | - |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
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