AVROBIO Inc., is a clinical stage gene therapy company that is on the path to develop potentially curative lentiviral-based gene therapies for rare genetic diseases known as lysosomal storage diseases. AVROBIO's approach is to use hemapoietic stem cells extracted from patients, modify these stems cells with lentiviral vectors, and insert a functional copy of the defective gene of the respective rare target disease. Today lysosomal storage diseases are managed by enzyme replacement therapies, also known as ERTs, and represent a substantial global market of approximately US$ 4 Billion in net sales (2017). AVROBIO's initial pipeline consists of 4 lentiviral-based gene therapies for the treatment of Fabry Disease, Gaucher Disease, Pompe Disease and Cystinosis.
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