Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DSC3P4
|
|||
| Drug Name |
mRNA-3927
|
|||
| Drug Type |
mRNA therapy
|
|||
| Indication | Propionic acidemia [ICD-11: 5C50.E0] | Phase 2 | [1] | |
| Company |
Moderna
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Propionyl-CoA carboxylase alpha chain, mitochondrial messenger RNA (PCCA mRNA) | Target Info | . | [2] |
| Propionyl-CoA carboxylase beta chain, mitochondrial messenger RNA (PCCB mRNA) | Target Info | . | [2] | |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT05130437) A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study. U.S.National Institutes of Health. | |||
| REF 2 | Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia. Nucleic Acid Ther. 2023 Apr;33(2):141-147. | |||
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