ADA-SCID
4
Pipeline Programs
6
Companies
2
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
4
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
6 companies ranked by most advanced pipeline stage
Genome & CompanyKorea - Suwon
1 program1
Infusion of autologous EFS-ADA LV CD34+Phase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Leadiant BiosciencesEZN-2279
Genome & CompanyInfusion of autologous EFS-ADA LV CD34+
Clinical Trials (2)
Total enrollment: 53 patients across 2 trials
EZN-2279 in Patients With ADA-SCID
Start: Jan 2014Est. completion: May 20197 patients
Phase 3Completed
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Start: Aug 2013Est. completion: Aug 201846 patients
Phase 1/2Completed
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.