Leukodystrophy, Globoid Cell
2
Pipeline Programs
2
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2022
2023
2024
2025
2026
2027
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2030
BioTherapeutics IncPBKR03
Clinical Trials (1)
Total enrollment: 24 patients across 1 trials
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
Start: Feb 2022Est. completion: Jan 203024 patients
Phase 1/2Suspended
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
2 companies competing in this space
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