SMN1 Gene Therapies
2 drugs2
Approved Drugs
1
Companies
0
Indications
0
Therapeutic Areas
SMN1 Drug Modality Landscape
Modalities
Gene therapy
2
100%
Routes of Administration
💉 IV
1
50%
💧 Other
1
50%
💡
SMN1 requires biologic approaches (gene therapy), likely due to its structure or location.
Biologics only
SMN1 Clinical Trials 8 trials
8
Total Trials
3
Active
4
Completed
80%
Completion Rate
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 0 | 1 | 0 | 0% |
| Phase 3 | 6 | 4 | 0 | 2 | 100% |
| Phase 4 | 1 | 0 | 0 | 1 | - |
Top Sponsors
Novartis Gene Therapies 6 80%
Novartis Pharmaceuticals 2
By Modality
Gene therapy 8 80%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)
SMN1 Drug Approval Timeline (2019 - 2025)
Modalities: Gene therapy | First of modality
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Competitive Landscape
- • 1 companies competing
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Full Drug Portfolio
- • All 2 approved drugs
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Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Success rates by condition
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