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

General Information of Drug (ID: DMIDEKB)

• Drug Name: AAV2-hFIX16 Drug Info
• Synonyms: AAV-F.IX, University of Pennsylvania; AAV-factor IX gene therapy (hemophilia B), University of Pennsylvania

Indication

Disease Entry	ICD 11	Status	REF
Factor IX deficiency	3B11	Phase 1	[1]

• Cross-matching ID: TTD Drug ID: DMIDEKB

Molecule(s) Related to This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Coagulation factor IX (F9)	TTFEZ5Q	FA9_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
Coagulation factor IX (F9)	DTT	F9	11.652	4.365	4.591	4.667

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

• The Studied Disease: Factor IX deficiency
• ICD Disease Classification: 3B11

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Coagulation factor IX (F9)	DTT	F9	5.69E-01	-0.13	-0.45

References

• [1] ClinicalTrials.gov (NCT00515710) LTFU for Gene Transfer Subjects With Hemophilia B. U.S. National Institutes of Health.
• [2] Assessing the potential for AAV vector genotoxicity in a murine model. Correction in: Blood. 2011 June 16; 117(24): 6739.