General Information of Drug (ID: DMUP52Y)

Drug Name
SB-FIX Drug Info
Indication
Disease Entry ICD 11 Status REF
Haemophilia B 3B11.0 Phase 1/2 [1]
Cross-matching ID
TTD Drug ID
DMUP52Y

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 [1]

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 (NCT02695160) Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B. U.S. National Institutes of Health.