Mucopolysaccharidosis Type II (MPS II)
2
Pipeline Programs
1
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
1
0
1
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
RegenxbioRGX-121
RegenxbioRGX-121
Clinical Trials (2)
Total enrollment: 54 patients across 2 trials
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
Start: Sep 2018Est. completion: Aug 202548 patients
Phase 2/3Active Not Recruiting
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
Start: Mar 2021Est. completion: May 20246 patients
Phase 1/2Completed
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 companies competing in this space
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