General Information of Drug (ID: DM7RNKM)

Drug Name
AMT-060 Drug Info
Synonyms AAV gene therapy (hemophilia B), Amsterdam Molecular Therapeutics/St Jude
Indication
Disease Entry ICD 11 Status REF
Factor IX deficiency 3B11 Phase 1/2 [1]
Cross-matching ID
TTD Drug ID
DM7RNKM

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
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 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
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800026919)
2 Phase I/II clinical trial of AMT-060 for treating hemophilia B. uniQure N.V.