Severe Combined Immunodeficiency Due to RAG1 Deficiency
1
Pipeline Programs
1
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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
1100%
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
GenewityGene therapy
Clinical Trials (1)
Total enrollment: 10 patients across 1 trials
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Start: Jul 2021Est. completion: Dec 203110 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
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.