Adenosine Deaminase 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
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
1 program1
Infusion of autologous EFS-ADA LV CD34+ cellsPhase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Orchard TherapeuticsInfusion of autologous EFS-ADA LV CD34+ cells
Clinical Trials (1)
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Start: Nov 2012Est. completion: Dec 2019
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 companies competing in this space