PARAMETER

DATA

Manufacturer

Orchard Therapeutics (Netherlands) BV

Transgene

-

Indication

Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)- matched related stem cell donor is available.

Virus and Serotype

Retroviral vector GSK3336223

Cell Substrate

Autologous genetically modified CD34+ cells

Manufacturing platform

Gene Therapy Vector (GSK3336223): The vector production process consists of thawing and expansion of the producer cell line, incubation with harvest medium and harvest. This is followed by filtration and freezing.

Transduced CD34+ cells with vector (GSK2696273): To generate the active substance, autologous CD34+ cells are isolated and transduced with the retroviral vector GSK3336223. The harvested bone marrow material of the patient is transferred from the syringe into another container and the number of CD34+ cells is assessed, and the cells undergo microbiological testing. Bone marrow aspirate is received into the facility, diluted and mononuclear cells (MNCs) are isolated for further processing. Cells are then washed, isolated and transduced with the GSK3336223 retroviral vector.

Dose in vial/final container

Strimvelis 1-10 million cells/ml dispersion for infusion.

Dose/patient

Strimvelis 1-10 million cells/ml dispersion for infusion.