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mRNA-3927 · 2 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| mRNA-3927 | EXPERIMENTAL | Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation. |
| Part 1 (Dose Optimization), Part 2 (Pivotal Study), and Part 3 (Infants) | EXPERIMENTAL | Part 1 (Dose Optimization): Participants (≥1 year of age) will receive single dose of mRNA-3927 by intravenous (IV) infusion every 2 weeks (Q2W) or every 3 weeks (Q3W) for up to 10 doses. Part 2 (Pivotal Study): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months. Part 3: Participants (\<1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months. |
| Name | Type | Description |
|---|---|---|
| mRNA-3927 | BIOLOGICAL | mRNA-3927 dispersion for IV infusion |
Inclusion Criteria: * Participated in Study mRNA-3927-P101. * Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study. Exclusion Criteria: * Not e...
mRNA-3927 is an investigational therapy being developed for propionic acidemia, a rare inherited metabolic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with this condition to assess its safety and clinical activity.
mRNA-3927 is being developed by Moderna, Inc., a biotechnology company publicly traded under the ticker symbol MRNA on the NASDAQ. Moderna is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for propionic acidemia.
mRNA-3927 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trials are designed to evaluate its safety, tolerability, and clinical activity in patients with propionic acidemia.
mRNA-3927 is being studied in two Phase 1 clinical trials. The first, NCT04159103, is an open-label study in participants with propionic acidemia. The second, NCT05130437, is a long-term safety and clinical activity study for participants previously enrolled in the first study. Both trials are currently recruiting.
mRNA-3927 is a messenger RNA (mRNA) therapy designed to address the underlying genetic cause of propionic acidemia. While the specific molecular target is not disclosed, the therapy aims to provide instructions for cells to produce a functional protein that is deficient in this condition, potentially restoring metabolic function.