An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

2019-08-13 18:05:44 | BioPortfolio


This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.

Study Design


Adenosine Deaminase Deficiency


autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)


Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles
United States


Enrolling by invitation


Orchard Therapeutics

Results (where available)

View Results


Published on BioPortfolio: 2019-08-13T18:05:44-0400

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Medical and Biotech [MESH] Definitions

Post-transcriptional regulatory proteins required for the accumulation of mRNAs that encode the gag and env gene products in HUMAN T-LYMPHOTROPIC VIRUS 1 and HUMAN T-LYMPHOTROPIC VIRUS 2. The rex (regulator x; x is undefined) products act by binding to elements in the LONG TERMINAL REPEAT.

The introduction of new genes into cells for the purpose of treating disease by restoring or adding gene expression. Techniques include insertion of retroviral vectors, transfection, homologous recombination, and injection of new genes into the nuclei of single cell embryos. The entire gene therapy process may consist of multiple steps. The new genes may be introduced into proliferating cells in vivo (e.g., bone marrow) or in vitro (e.g., fibroblast cultures) and the modified cells transferred to the site where the gene expression is required. Gene therapy may be particularly useful for treating enzyme deficiency diseases, hemoglobinopathies, and leukemias and may also prove useful in restoring drug sensitivity, particularly for leukemia.

Proteins coded by the retroviral gag gene. The products are usually synthesized as protein precursors or POLYPROTEINS, which are then cleaved by viral proteases to yield the final products. Many of the final products are associated with the nucleoprotein core of the virion. gag is short for group-specific antigen.

Proteins encoded by the POL GENE of the HUMAN IMMUNODEFICIENCY VIRUS.

Proteins encoded by the ENV GENE of the HUMAN IMMUNODEFICIENCY VIRUS.

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