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Krabbe Disease

0
Pipeline Programs
4
Companies
5
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
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Forge Biologics
Forge BiologicsOH - Grove City
3 programs
FBX-101N/A1 trial
FBX-101PHASE_1_21 trial
FBX-101PHASE_1_21 trial
Active Trials
NCT06308718Terminated2Est. Apr 2025
NCT04693598Active Not Recruiting6Est. Nov 2026
NCT05739643Active Not Recruiting9Est. Nov 2026
CENTOGENE
CENTOGENEGermany - Rostock
1 program
Biomarker for Krabbe Disease (BioKrabbe)N/A1 trial
Active Trials
NCT01425489Withdrawn0Est. Feb 2021
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
Krabbe Disease Global Patient RegistryN/A
Rare Disease Therapeutics
1 program
Krabbe Disease Global Patient RegistryN/A1 trial
Active Trials
NCT02993796Recruiting60Est. Sep 2026

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Forge BiologicsFBX-101
Forge BiologicsFBX-101
Forge BiologicsFBX-101
CENTOGENEBiomarker for Krabbe Disease (BioKrabbe)
Rare Disease TherapeuticsKrabbe Disease Global Patient Registry

Clinical Trials (5)

Total enrollment: 77 patients across 5 trials

NCT05739643Forge BiologicsFBX-101

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

Start: Feb 2023Est. completion: Nov 20269 patients
Phase 1/2Active Not Recruiting
NCT04693598Forge BiologicsFBX-101

Gene Transfer Clinical Trial for Krabbe Disease

Start: Nov 2021Est. completion: Nov 20266 patients
Phase 1/2Active Not Recruiting
NCT06308718Forge BiologicsFBX-101

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Start: Aug 2024Est. completion: Apr 20252 patients
N/ATerminated
NCT01425489CENTOGENEBiomarker for Krabbe Disease (BioKrabbe)

Biomarker for Krabbe Disease (BioKrabbe)

Start: Aug 2018Est. completion: Feb 20210
N/AWithdrawn
NCT02993796Rare Disease TherapeuticsKrabbe Disease Global Patient Registry

Krabbe Disease Global Patient Registry

Start: Sep 2014Est. completion: Sep 202660 patients
N/ARecruiting

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 77 patients
4 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.