Severe Combined Immunodeficiency Due to ADA Deficiency
2
Pipeline Programs
2
Companies
3
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
1
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Angeles TherapeuticsCA - Los Angeles
2 programs1
1
OTL-101Phase 2/31 trial
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cellsPhase 1/21 trial
Active Trials
Orchard TherapeuticsMA - Boston
2 programsInfusion of autologous cryopreserved EFS-ADA LV CD34+ cellsPHASE_1_21 trial
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cellsPHASE_1_2
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Angeles TherapeuticsOTL-101
Orchard TherapeuticsInfusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Angeles TherapeuticsInfusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Clinical Trials (3)
Total enrollment: 23 patients across 3 trials
A Clinical Study to Enable Process Validation of Commercial Grade OTL-101
Start: Oct 2019Est. completion: Aug 20210
Phase 2/3Withdrawn
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Start: Jan 2018Est. completion: Sep 202213 patients
Phase 1/2Completed
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Start: Dec 2016Est. completion: Sep 201910 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
2 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.