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

General Information of Drug (ID: DMMKE2H)

Drug Name
Valoctocogene roxaparvovec Drug Info
Synonyms BMN 270
Indication
Disease Entry ICD 11 Status REF
Haemophilia A 3B10.0 Phase 3 [1]
Cross-matching ID
TTD Drug ID
DMMKE2H

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Coagulation factor VIII (F8) TT1290U FA8_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 VIII (F8) DTT F8 7.637 6.9 7.644 6.975
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 A
ICD Disease Classification 3B10.0
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Coagulation factor VIII (F8) DTT F8 1.08E-22 0.89 1.82
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT04323098) Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3). U.S. National Institutes of Health.
2 Clinical pipeline report, company report or official report of BioMarin Pharmaceutical.