Lysosomal Storage Diseases
1
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
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
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2031
Orchard TherapeuticsOTL-200
CENTOGENELyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
Clinical Trials (2)
Total enrollment: 299 patients across 2 trials
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Start: Jan 2022Est. completion: Mar 2031
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
1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space