General Information of Drug (ID: DMURO94)

Drug Name
GNE Lipoplex Drug Info
Synonyms GNE Lipoplex (intramuscular, HIBM-2)
Indication
Disease Entry ICD 11 Status REF
Myopathy 8C7Y Phase 1 [1]
Cross-matching ID
TTD Drug ID
DMURO94

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
N-acetylmannosamine kinase (GNE) TT4DP5S GLCNE_HUMAN Inhibitor [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
N-acetylmannosamine kinase (GNE) DTT GNE 8.942 8.103 6.71 5.558
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

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

ICD Disease Classification 08 Disease of the nervous system
Disease Class ICD-11: 8A00 Parkinsonism
The Studied Tissue Muscle tissue
The Studied Disease Myopathy [ICD-11:8A00.0]
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
N-acetylmannosamine kinase (GNE) DTT GNE 7.10E-01 -0.02 -0.04
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 800034673)
2 Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex. Hum Gene Ther. 2011 Nov;22(11):1331-41.