Details of the Drug-Related molecule(s) Expression Atlas

General Information of Drug (ID: DMMKASG)

Drug Name
AGTC-0106 Drug Info
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 Drug ID
DMMKASG

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Alpha-1-antitrypsin (SERPINA1) TTA7UJC A1AT_HUMAN Modulator [2]

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule Molecule Type Gene Name Liver Colon Kidney Small Intestine
Alpha-1-antitrypsin (SERPINA1) DTT SERPINA1 12.865 6.98 11.766 10.09
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

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 between Disease Section and Healthy Individual Tissue

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.