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AVROBIO, Inc. is a clinical-stage gene therapy company. The Company is focused on developing curative ex vivo lentiviral-based gene therapies to treat patients with rare diseases following a single dose treatment regimen. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The Company is focusing on a group of rare genetic diseases referred to as lysosomal disorders, primarily managed with enzyme replacement therapies. Its pipeline is comprised of various HSC gene therapy programs, which include AVR-RD-02 for the treatment of Gaucher disease type 1 and type 3; AVR-RD-05 for the treatment of neuronopathic mucopolysaccharidosis type II (MPS-II) or Hunter syndrome; and AVR-RD-03 for the treatment of Pompe disease.
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