SOD1 mRNA Inhibitors
1 drugsAbout SOD1 mRNA
Superoxide dismutase 1 (SOD1) mRNA encodes the superoxide dismutase 1 enzyme, protecting cells from damaging free radicals. Aberrant SOD1 mRNA expression can disrupt cellular homeostasis, potentially contributing to disease pathology, especially within the central nervous system (CNS). Targeting SOD1 mRNA allows modulation of protein levels by interfering with the messenger RNA.
There is currently no genetic evidence directly linking SOD1 mRNA levels to specific diseases. However, the approval of QALSODY underscores the therapeutic potential of targeting SOD1 mRNA. Reducing SOD1 production may be beneficial in conditions where aberrant expression contributes to disease.
SOD1 mRNA is targeted by one FDA-approved drug, QALSODY, developed by Biogen for CNS disorders. QALSODY is an antisense oligonucleotide (ASO), highlighting the feasibility of this modality for therapeutic intervention. Further research may explore additional drug modalities.
Top SOD1 mRNA Drugs
Biogen is currently the only company with an approved drug targeting SOD1 mRNA.
High market concentration suggests potential for new entrants, but also high barriers to entry.
SOD1 mRNA Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets SOD1 mRNA, using aso modality.
Explore alternative modalities to differentiate from existing therapies and expand market reach.
SOD1 mRNA Clinical Trials 6 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on SOD1 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 | 0 | 0 | 1 | - |
| Phase 3 | 3 | 2 | 0 | 1 | 100% |
| Phase 4 | 1 | 0 | 0 | 1 | - |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
1 Phase 3 trial testing approved SOD1 mRNA drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting SOD1 mRNA. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
SOD1 mRNA Drug Approval Timeline (2023 - 2023)
QALSODY was the first and most recent drug approved in 2023, spanning a 1-year approval history.
Recent approval indicates growing interest, but also suggests limited clinical validation and market saturation.
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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 (1 company)
- • 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 SOD1 mRNA. A trial counts if an approved drug acting on SOD1 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.