G1XCGD

G1XCGD is a self-inactivating lentiviral vector developed for ex vivo gene therapy targeting X-linked Chronic Granulomatous Disease (X-CGD). This gene therapy involves transducing a patient's own CD34+ hematopoietic stem and progenitor cells with the G1XCGD vector, which carries a codon-optimized human CYBB cDNA encoding for the gp91phox subunit of NADPH oxidase. The goal is to restore functional NADPH oxidase activity in phagocytes, thereby enhancing the immune system's ability to combat severe bacterial and fungal infections characteristic of X-CGD. Initially developed by Genethon, the program was later licensed by Orchard Therapeutics, which designated it OTL-102. Kyowa Kirin subsequently acquired Orchard Therapeutics. G1XCGD has received Orphan Drug Designation in both Europe and the United States and is currently undergoing Phase I/II clinical trials.

Other names

OTL-102OTL102OTL 102