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]
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Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

Molecular Interaction Atlas (MIA)

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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)

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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.