Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)

GENETIC2 trials

Sponsors

University of California, Los Angeles, Great Ormond Street Hospital for Children NHS Foundation Trust

Conditions

Severe Combined Immunodeficiency Due to ADA Deficiency

Phase 1

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

CompletedNCT02999984

University of California, Los AngelesSevere Combined Immunodeficiency Due to ADA Deficiency

Start: 2016-12-16End: 2019-09-26Updated: 2022-08-03

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

CompletedNCT03765632

Great Ormond Street Hospital for Children NHS Foundation TrustSevere Combined Immunodeficiency Due to ADA Deficiency

Start: 2018-01-03End: 2022-09-28Updated: 2023-02-01

Related Papers

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Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood2019-11-1314 citations