Lysosomal Storage Disease
0
Pipeline Programs
3
Companies
4
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
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
1100%
+ 3 programs with unclassified modality
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
2 programsOTL-200 Gene TherapyPHASE_1_2Gene Therapy1 trial
OTL-200PHASE_21 trial
Active Trials
CENTOGENEGermany - Rostock
1 programBiomarker for Maroteaux-Lamy Disease (BioMaroteaux)N/A1 trial
Active Trials
Human BioSciencesWV - Martinsburg
1 programStudy of Inborn Errors of Cholesterol Synthesis and Related DisordersN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Orchard TherapeuticsOTL-200
Orchard TherapeuticsOTL-200 Gene Therapy
CENTOGENEBiomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Human BioSciencesStudy of Inborn Errors of Cholesterol Synthesis and Related Disorders
Clinical Trials (4)
Total enrollment: 372 patients across 4 trials
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Start: Jan 2018Est. completion: Mar 202610 patients
Phase 2Completed
Gene Therapy for Metachromatic Leukodystrophy (MLD)
Start: Apr 2010Est. completion: Sep 202520 patients
Phase 1/2Completed
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Start: Aug 2018Est. completion: Feb 20210
N/AWithdrawn
Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
Start: Oct 2002342 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
3 companies competing in this space
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