NGLY1 Deficiency
1
Pipeline Programs
1
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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Grace ScienceCA - Menlo Park
2 programs1
GS-100Phase 1/21 trial
NGLY1 Natural HistoryN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2023
2024
2025
2026
2027
2028
Grace ScienceGS-100
Grace ScienceNGLY1 Natural History
Clinical Trials (2)
Total enrollment: 21 patients across 2 trials
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
Start: Feb 2024Est. completion: Jan 20286 patients
Phase 1/2Recruiting
NGLY1 Natural History
Start: Feb 2023Est. completion: Oct 202415 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
1 actively recruiting trials targeting 21 patients
1 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.