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Drugs / SIMOLADAGENE AUTOTEMCEL

Follow last change Sep 2022 re-read 2 minutes ago

SIMOLADAGENE AUTOTEMCEL

also known as OTL-101 · Simoladagen autotemcel

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: Orchard Therapeutics · Great Ormond Street Hospital for Children NHS Foundation Trust · University of California, Los Angeles · California Institute for Regenerative Medicine (CIRM)

Trials 4 · a red edge is where a trial was stopped

today NCT04140539 · Ph 2/3 NCT03765632 · Ph 1/2 NCT02999984 · Ph 1/2 NCT01380990 · Ph 1/2

201320142015201620172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 1/23 trials
Phase 1/2	NCT03765632	Jan 2018 → Sep 2021	severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency	Great Ormond Street Hospital for Children NHS Foundation Trust	Completed	No outcome recorded
Phase 1/2	NCT02999984	Dec 2016 → Oct 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
Phase 1/2	NCT01380990	Nov 2012 → Dec 2019	severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency	Great Ormond Street Hospital for Children NHS Foundation Trust	Completed	No outcome recorded
Phase 2/31 trial
Phase 2/3	NCT04140539	Oct 2019 → Aug 2021	severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency	University of California, Los Angeles	Withdrawn	No outcome recorded

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 5 sources stand behind the page.

Field	Value	Cited text
Known as	SIMOLADAGENE AUTOTEMCEL	ChEMBL registry synonym — accepted as the source's own label CHEMBL4298201 ↗
Known as	OTL-101	“Autologous EFS-ADA LV CD34+ cells (OTL-101*) are infused intravenously” NCT01380990 ↗ 4 “OTL-101” NCT03765632 ↗ NCT03765632 ↗ NCT02999984 ↗ NCT04140539 ↗
Known as	Simoladagen autotemcel	ChEMBL registry synonym — accepted as the source's own label CHEMBL4298201 ↗
Route	Intravenous	“are infused intravenously” NCT01380990 ↗
Target	ADA	“EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene” NCT02999984 ↗