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Rare Disease
Spinal Muscular Atrophy
12
Pipeline Programs↗
34
Active Trials↗
15
Companies Involved↗
Spinal Muscular Atrophy is an emerging landscape with 12 programs across 15 companies. The most active players are Novartis, Biogen, Roche. 7 programs are in Phase 3.
Intelligence
Pipeline Metrics
Competitive Density
Pipeline Velocity
Mech. Concentration
Completion Rate
Attrition Rate
Competition
Competitive Landscape
Bubble size = clinical trials
Approved
Phase 3
Phase 2
Phase 1
Preclinical
Key Players
Top Companies Companies ranked by the number of pipeline programs they run for this disease.
Pipeline
Programs by Phase
Preclinical2
Phase 1
No programs
Phase 37
Approved
No programs
Analysis
Modality Breakdown
9programs
Small molecule556%
Gene therapy222%
Monoclonal antibody111%
RNA therapy111%
Modality classified for 92% of 12 pipeline programs.
Analysis
Trial Status
87
clinical trials
Completed39 · 45%
Recruiting15 · 17%
Active, not recruiting12 · 14%
Terminated8 · 9%
Not yet recruiting5 · 6%
Enrolling by invitation2 · 2%
Other6 · 7%
Clinical Trials
Trial Activity
87 trials
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)