autologous CD34+ cells transduced with the G1XCGD lentiviral vector

Autologous CD34+ cells transduced with the G1XCGD lentiviral vector (also known as OTL-102) is an ex vivo gene therapy developed by Genethon, with Orchard Therapeutics previously holding an exclusive licensing option. The therapy consists of the patient's own autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with the G1XCGD lentiviral vector. This self-inactivating lentiviral vector carries a codon-optimized human CYBB gene encoding the gp91phox (NOX2) protein, which is the catalytic subunit of the NADPH oxidase complex. The therapy is designed to treat X-linked chronic granulomatous disease (X-CGD), a rare inherited immunodeficiency where mutations in CYBB prevent phagocytes from producing reactive oxygen species to kill pathogens. By restoring functional gp91phox expression in myeloid progeny cells, the therapy aims to reconstitute NADPH oxidase activity and immune function. Clinical trials (including NCT01855685, NCT02757911, and NCT02234934) have evaluated its safety and efficacy, though some trials have been terminated or completed, and Orchard Therapeutics discontinued its development in 2022.

Other names

G1XCGDG-1XCGDG 1XCGDG1XCGD Modified Autologous BM CD34 cellsG-1XCGD Modified Autologous BM CD34 cellsG 1XCGD Modified Autologous BM CD34 cellspCCLchimGP91WPRE transduced CD34+ cells