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Drugs / EFS-ADA LV CD34+ cells
last change May 2021 re-read 2 minutes ago

EFS-ADA LV CD34+ cells

targets ADA

Developed for
severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency
Investigated by
National Heart, Lung, and Blood Institute (NHLBI) · National Human Genome Research Institute (NHGRI) · National Institute of Allergy and Infectious Diseases (NIAID) · Orchard Therapeutics · University of California, Los Angeles

Trials 1 · a red edge is where a trial was stopped

today NCT01852071 · Ph 1/2
20142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT01852071 Aug 2013 → Aug 2018 severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency University of California, Los Angeles Completed No outcome recorded

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as EFS-ADA LV CD34+ cells “autologous EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously” NCT01852071 ↗
Route Intravenous “are infused intravenously” NCT01852071 ↗
Target ADA “lentiviral vector encoding human ADA” PMID 33974366 ↗ May 2021