Severe Combined Immunodeficiency Due to ADA Deficiency
2
Pipeline Programs
1
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
2 programs2
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cellsPhase 1/21 trial
Infusion of autologous cryopreserved 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 cryopreserved EFS-ADA LV CD34+ cells
Orchard TherapeuticsInfusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Clinical Trials (2)
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Start: Jan 2018Est. completion: Sep 2022
Phase 1/2Completed
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Start: Dec 2016Est. completion: Sep 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