AT mRNA Inhibitors
1 drugsAbout AT mRNA
Adenine Translocase mRNA (AT mRNA) is a drug target currently being investigated for potential therapeutic interventions. Research is ongoing to fully elucidate its role in disease pathology and its mechanism of action.
Currently, the therapeutic application of AT mRNA modulation is focused on rare diseases. There is no genetic evidence data available linking AT mRNA to specific diseases, highlighting the need for further research.
QFITLIA (Sanofi) is the only FDA-approved drug targeting AT mRNA, indicated for rare diseases. It is an siRNA-based therapy, representing 100% of the modality landscape for this target.
Top AT mRNA Drugs
Sanofi is the only company with an approved drug targeting AT mRNA.
The lack of competitors suggests a high barrier to entry or an unproven market.
AT mRNA Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets AT mRNA, using sirna modality.
Exploring alternative modalities could provide a competitive advantage in this space.
AT mRNA Clinical Trials 9 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on AT mRNA. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 2 | 1 | 1 | 0 | 0 | 100% |
| Phase 3 | 6 | 4 | 0 | 2 | 100% |
| Phase 4 | 1 | 0 | 0 | 1 | - |
Top Sponsors
By Modality
Top Conditions
Top Drugs
AT mRNA Drug Approval Timeline (2025 - 2025)
The first and only drug targeting AT mRNA, QFITLIA, was approved in 2025.
The recent approval suggests a growing interest in AT mRNA as a therapeutic target.
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Deep insights for drug target analysis
Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 2 (2 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on AT mRNA. A trial counts if an approved drug acting on AT mRNA is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.