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Mucopolysaccharidosis III-B

1
Pipeline Programs
2
Companies
3
Clinical Trials
1 recruiting
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

2 companies ranked by most advanced pipeline stage

JCR Pharmaceuticals
JCR PharmaceuticalsJapan - Hyogo
1 program
1
JR-446Phase 1/22 trials
Active Trials
NCT07640984Not Yet Recruiting12Est. Sep 2028
NCT06488924Recruiting10Est. Apr 2030
Abeona Therapeutics
Abeona TherapeuticsCLEVELAND, OH
1 program
ABO-101N/A1 trial
Active Trials
NCT04655911Terminated1Est. Apr 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
JCR PharmaceuticalsJR-446
JCR PharmaceuticalsJR-446
Abeona TherapeuticsABO-101

Clinical Trials (3)

Total enrollment: 23 patients across 3 trials

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

Start: Jun 2026Est. completion: Sep 202812 patients
Phase 1/2Not Yet Recruiting

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Start: Nov 2024Est. completion: Apr 203010 patients
Phase 1/2Recruiting

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Start: Oct 2020Est. completion: Apr 20221 patients
N/ATerminated

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 23 patients
2 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.