AVR-RD-06

AVR-RD-06 is an investigational ex vivo lentiviral hematopoietic stem cell (HSC) gene therapy being developed by Avrobio for the treatment of Gaucher disease type 3, the chronic neuronopathic form of Gaucher disease. It uses a lentiviral vector encoding a codon-optimized human glucocerebrosidase (GCase) gene, designed to stably integrate into autologous CD34+ HSCs and enable long-term systemic and CNS-relevant production of functional GCase after reinfusion.[2][13] By restoring GCase activity in cells of the monocyte–macrophage lineage, AVR-RD-06 aims to reduce pathological accumulation of glucosylceramide and related substrates and address both systemic and neurologic manifestations of Gaucher disease type 3.[2][13]