A New Era of Medicine
Gene therapy represents a new paradigm in human health, with the potential to resolve unmet medical needs by addressing the underlying causes of disease.
AVROBIO, Inc., a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose. The Company is focused on the development of its clinical stage programs in Fabry disease and acute myeloid leukemia (AML), while actively building a pipeline of gene therapies to treat Gaucher disease and other rare diseases.
Gene therapies have been shown to deliver dramatic disease-modifying effects and potentially life-long cures from a single infusion of genetically-modified cells. Underpinning these advances are a deep understanding of cell biology, immunology and advanced vector designs enabling safe and effective delivery of therapeutic genes targeted to specific cells.