autologous CD34+ cells transduced with lentiviral IDS.ApoEII

Autologous CD34+ cells transduced with lentiviral IDS.ApoEII (also known as AVR-RD-05) is an investigational ex vivo gene therapy developed by the University of Manchester (previously in collaboration with AVROBIO) for the treatment of neuronopathic Mucopolysaccharidosis type II (MPS II, Hunter Syndrome). The therapy involves harvesting the patient's own (autologous) CD34+ hematopoietic stem cells, which are then genetically modified ex vivo using a self-inactivating (SIN) lentiviral vector (CD11b.IDS-ApoEII LV) encoding a human codon-optimized iduronate-2-sulfatase (IDS) gene tagged with ApoEII, driven by a human CD11b myeloid-specific promoter. The ApoEII tag is specifically designed to enhance CNS targeting and delivery of the IDS enzyme across the blood-brain barrier via receptor-mediated transcytosis. Once infused back into the patient, the modified stem cells repopulate the blood system and produce functional IDS enzyme, which can cross-correct affected cells in somatic organs and the central nervous system. It is currently being evaluated in a Phase I/II clinical trial (NCT05665166) in male infants with neuronopathic-risk MPS II.

Other names

autologous CD34+ cells transduced with lentiviral IDS.ApoEIICD11b.IDS-ApoEII LV transduced autologous CD34+ cellsautologous CD34+ hematopoietic stem cells transduced ex vivo with CD11B lentiviral vector encoding human IDS tagged with ApoEII