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Batten Disease

2
Pipeline Programs
3
Companies
3
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Alcyone Therapeutics
1 program
1
CLN-301Phase 1/21 trial
Active Trials
NCT03770572Active Not Recruiting7Est. Sep 2029
Theranexus
TheranexusFrance - Lyon
1 program
1
Miglustat 100 milligramsPhase 1/21 trial
Active Trials
NCT05174039Completed6Est. May 2024
BioMarin Pharmaceutical
1 program
CLN2 TreatmentN/A1 trial
Active Trials
NCT03862274Enrolling By Invitation30Est. Dec 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
TheranexusMiglustat 100 milligrams
Alcyone TherapeuticsCLN-301
BioMarin PharmaceuticalCLN2 Treatment

Clinical Trials (3)

Total enrollment: 43 patients across 3 trials

NCT05174039TheranexusMiglustat 100 milligrams

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

Start: Mar 2022Est. completion: May 20246 patients
Phase 1/2Completed
NCT03770572Alcyone TherapeuticsCLN-301

Gene Therapy for Children With CLN3 Batten Disease

Start: Nov 2018Est. completion: Sep 20297 patients
Phase 1/2Active Not Recruiting
NCT03862274BioMarin PharmaceuticalCLN2 Treatment

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Start: Dec 2018Est. completion: Dec 202530 patients
N/AEnrolling By Invitation

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

3 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.