GM1 Gangliosidosis
2
Pipeline Programs
6
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
5 companies ranked by most advanced pipeline stage
AzafarosNetherlands - DC Naarden
1 programGM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)N/A1 trial
Active Trials
BiocorpFrance - Issoire
1 programNatural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) PatientsN/A1 trial
Active Trials
Passage BioPA - Philadelphia
1 programNatural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) PatientsN/A
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
GEMMA BiotherapeuticsPBGM01
AzafarosGM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
BiocorpNatural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Clinical Trials (3)
Total enrollment: 88 patients across 3 trials
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
Start: Mar 2021Est. completion: Feb 202926 patients
Phase 1/2Active Not Recruiting
NCT05109793AzafarosGM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
Start: Feb 2022Est. completion: Sep 202531 patients
N/ACompleted
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Start: Jun 2020Est. completion: May 202431 patients
N/ACompleted
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
6 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.