Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)

NCTID	NCT05071222 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA. (Show More)
Indication	Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency
Compound Name	EF1a-hArtemis LV (modified EF1a promotor) ARTEGENE
Sponsor	Assistance Publique - Hôpitaux de Paris
Funder Type	Other
Status	Recruiting
Enrollment Count	5

Target Gene/Variant	DCLRE1C
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Transduced CD34+ cells, range: 1.8-3.7E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	France

Has US IND	False
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