Sanfilippo Syndrome (MPS III)
1
Pipeline Programs
1
Companies
1
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
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2026
2027
2028
2029
NeuroGTrAAV9-CMV-hNAGLUop
Clinical Trials (1)
Total enrollment: 9 patients across 1 trials
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
Start: Jan 2027Est. completion: Dec 20299 patients
Phase 1/2Not Yet Recruiting
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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