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
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on SMN1. Source: ClinicalTrials.gov. How we count
8
Total Trials
3
Active
4
Completed
80%
Completion Rate
Completion by Phase
| Phase | Total | Completed | Stopped | 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Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)
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Competitive Landscape
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- • All 2 approved drugs
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Approval Timeline
- • Full 2-drug timeline
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Clinical Trials Analysis
- • Sponsors: 2 (2 companies)
- • Completion rates by condition
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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 SMN1. A trial counts if an approved drug acting on SMN1 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- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn.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- Stopped: terminated or withdrawn.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.