MPS IIIA
4
Pipeline Programs
2
Companies
2
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
2
0
2
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Abeona TherapeuticsCLEVELAND, OH
2 programs1
1
UX111Phase 2/31 trial
ABO-102Phase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Abeona TherapeuticsUX111
Abeona TherapeuticsABO-102
Clinical Trials (2)
Total enrollment: 41 patients across 2 trials
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Start: Apr 2016Est. completion: Jul 202736 patients
Phase 2/3Recruiting
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Start: Sep 2019Est. completion: Mar 20225 patients
Phase 1/2Terminated
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 actively recruiting trials targeting 41 patients
2 companies competing in this space