MPS II
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
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
RegenxbioRGX-121-3102
GC BiopharmaHunterase
Clinical Trials (2)
Total enrollment: 32 patients across 2 trials
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
Start: Nov 2025Est. completion: May 20312 patients
Phase 3Suspended
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
Start: May 2018Est. completion: Dec 202230 patients
Phase 3Completed
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
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