Lysosomal Storage Diseases
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Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
CENTOGENEGermany - Rostock
1 programLyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher DiseaseN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
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Orchard TherapeuticsOTL-200
CENTOGENELyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
Clinical Trials (2)
Total enrollment: 305 patients across 2 trials
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Start: Jan 2022Est. completion: Mar 20316 patients
Phase 3Active Not Recruiting
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
Start: Aug 2018Est. completion: Jan 2021299 patients
N/ACompleted
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
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