Adenosine Deaminase Deficiency
2
Pipeline Programs
4
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
2
0
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
1 programInfusion of autologous EFS-ADA LV CD34+ cellsPHASE_1_21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
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2026
Orchard TherapeuticsInfusion of autologous EFS-ADA LV CD34+ cells
Genome & CompanyLentiviral Gene Transfer
Clinical Trials (2)
Total enrollment: 37 patients across 2 trials
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Start: Nov 2012Est. completion: Dec 201936 patients
Phase 1/2Completed
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector
Start: Dec 2013Est. completion: Sep 20171 patients
Phase 1Completed
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
4 companies competing in this space
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