AVROBIO

Cellular & Gene Therapies

Also Clin0 Gaucher disease type 3; AVR-RD-04 Clin1/Clin2 cystinosis; AVR-RD-05 Clin0 Hunter syndrome; AVR-RD-03 Clin0 Pompe disease

AVROBIO is reversing lysosomal disorders by developing lentiviral-based gene therapies. The current standard of care for treating lysosomal disorders is enzyme replacement therapy which slows, but does not halt overall disease progression. AVROBIO is creating investigational therapies designed to enable patients’ own bodies to begin producing the functional protein they lack. The therapies AVROBIO is developing use lentiviral vectors to deliver therapeutic genes to patient’s hematopoietic stem cells. AVROBIO’s approach is designed to enable the genetically modified cells to differentiate into different cell types, including monocytes which can then enter the central nervous system. Currently, AVROBIO has investigational therapies in clinical studies for Gaucher disease type 1 and cystinosis.