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Pompe disease is a lysosomal disorder caused by a mutation in the GAA gene. The lack of the enzyme encoded by GAA results in a toxic buildup of glycogen throughout the body and central nervous system.
The results demonstrate that gene therapy using hematopoietic stem cells (HSC) significantly reduced the toxic accumulation of glycogen in a mouse model of Pompe disease, including in cardiac and skeletal muscle and the central nervous system (CNS).
Eight months post-infusion, substrate levels in multiple treated tissues were nearly indistinguishable from normal mice.
Additionally, new data from a separate study show that human HSCs were efficiently transduced, producing robust quantities of transgene product and GAA enzyme activity.
AVROBIO plans to engage with regulatory agencies on the clinical development strategy for AVR-RD-03 in 2022 and plans to initiate a clinical trial in 2023.
Price Action: AVRO shares are down 4.38% at $0.79 during the market session on the last check Wednesday.
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