Details of the Drug

General Information of Drug (ID: DMMKASG)

Drug Name
AGTC-0106
Synonyms Alpha-1 antitrypsin deficiency gene therapy, AGTC; RAAV1-CB-hAAT; Gene therapy (alpha-1 antitrypsin deficiency), Applied Genetic Technologies
Indication
Disease Entry ICD 11 Status REF
Alpha-1 antitrypsin deficiency 5C5A Phase 2 [1]
Cross-matching ID
TTD ID
D0QF2W

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Alpha-1-antitrypsin (SERPINA1) TTA7UJC A1AT_HUMAN Modulator [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

The Studied Disease Alpha-1 antitrypsin deficiency
ICD Disease Classification 5C5A
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Alpha-1-antitrypsin (SERPINA1) DTT SERPINA1 7.84E-01 -0.08 -0.1
Alpha-1-antitrypsin (SERPINA1) DTT SERPINA1 1.32E-03 0.33 0.54
Molecular Expression Atlas (MEA) Jump to Detail Molecular Expression Atlas of This Drug

References

1 ClinicalTrials.gov (NCT01054339) Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. U.S. National Institutes of Health.
2 First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9.