ADA-SCID
2
Pipeline Programs
2
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
1
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
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
Orchard TherapeuticsInfusion of autologous EFS-ADA LV CD34+
Clinical Trials (2)
Total enrollment: 7 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 2018
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
1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space