Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

NCTID	NCT05432310 (View at clinicaltrials.gov)
Description	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints. (Show More)
Development Status	Active
Indication	Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Disease Ontology Term	DOID:5810
Compound Name	Simoladagene autotemcel
Compound Alias	OTL-101
Sponsor	University of California, Los Angeles
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	20
Results Posted	Not Available

Target Gene/Variant	ADA
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, minimum dose: Fresh (1E6 cells/kg); Cryopreserved (2E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Orchard terminated this program, returned the program to UCLA which administers the therapy under Compassionate Use