PEG-ADA ERT

DRUG2 trials

Sponsors

University of California, Los Angeles

Conditions

ADA-SCIDSevere Combined Immunodeficiency Due to ADA Deficiency

Phase 1

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

CompletedNCT01852071

University of California, Los AngelesADA-SCID

Start: 2013-08-02End: 2018-08-27Updated: 2022-08-03

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

Related Papers

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

New England Journal of Medicine2021-05-11204 citations

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

Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency

Biology of Blood and Marrow Transplantation2015-01-22