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

General Information of Drug (ID: DMFZ81T)

Drug Name
Fidanacogene elaparvovec Drug Info
Synonyms AAV8-hFIX19
Indication
Disease Entry ICD 11 Status REF
Haemophilia B 3B11.0 Phase 3 [1]
Cross-matching ID
TTD Drug ID
DMFZ81T

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 Replacement [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
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 Haemophilia B
ICD Disease Classification 3B11.0
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
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT03861273) A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2). U.S. National Institutes of Health.
2 Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua. J Thromb Haemost. 2021 May;19(5):1212-1218.