Autologous lentiviral G1XCGD gene therapy

Unassigned

New Medicines

X-linked chronic granulomatous disease (CGD)

Information

Advanced therapy medicinal product (ATMP)
Orchard Therapeutics
Orchard Therapeutics

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes

Category

Autologous CD34+ cells transduced with lentiviral vector containing XCGD gene
CGD is a primary immunodeficiency disorder of phagocytes (neutrophils, monocytes, macrophages and eosinophils) resulting from impaired killing of bacteria and fungi. CGD is rare (prevalence approximately 1 in 250,000). CGD is caused by pathogenic variants in one of five genes that encode the subunits of phagocyte NADPH oxidase: biallelic pathogenic variants in CYBA, NCF1, NCF2 and NCF4 cause autosomal recessive CGD (AR-CGD), and mutation of CYBB causes X-linked CGD [1]
X-linked chronic granulomatous disease (CGD)
Intravenous infusion