Lysosomal Storage Disease
2
Pipeline Programs
2
Companies
3
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
1
1
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
1100%
+ 2 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
2 programs1
1
OTL-200Phase 21 trial
OTL-200 Gene TherapyPhase 1/2Gene Therapy1 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)
Clinical Trials (3)
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Start: Jan 2018Est. completion: Jan 2026
Phase 2Active Not Recruiting
Gene Therapy for Metachromatic Leukodystrophy (MLD)
Start: Apr 2010Est. completion: Sep 2025
Phase 1/2Completed
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Start: Aug 2018Est. completion: Feb 20210
N/AWithdrawn
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