We develop products and processes for ex vivo gene therapy using lentiviral vectors and CRISPR genome editing technology.
Cell processing is generally required to prepare cells for ex vivo gene-modification. Cell processing steps include the separation of blood components, magnetic bead cell sorting and cryopreservation.
Advanced-generation recombinant HIV-1-derived lentiviral vectors are used for gene transfer or for genome editing. ART-TG has R&D expertise in LV bioproduction, particularly the development of downstream processes for the purification of LV with various envelope pseudotypes.
CRISPR Cas9 tools are used for genome editing. Synthetic or vectorized constructs are used and are delivered into cells with various methods including electroporation or infection.
Specific quality controls are used to analyze gene-modified cells or materials and include the quantification of genome integrated vector copies, the caracterization of genomic insertion sites, potency assays, infectious and physical titers.