A clinical-stage gene therapy company striving to halt or reverse
disease with a single dose.
We are AVROBIO
Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by its industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
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Genetically modified hematopoietic stem cells lead to supraphysiological levels of therapeutic protein in a mouse model of Pompe disease sustained at eight months after administration Substantial reduction in glycogen observed across cardiac and skeletal muscles, as well as CNS Data support plans
New early data show key visual motor integration, visual perception and motor coordination measures impacted by cystinosis stabilized or improved post gene therapy Systemic reach of AVR-RD-04 observed across multiple other measures All five dosed patients remain off oral cysteamine post gene
CAMBRIDGE, Mass. --(BUSINESS WIRE)--May 16, 2022-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced members of its senior management team will be presenting virtually at the H.C.