Avrobio's Pompe Disease Gene Therapy Shows Efficacy, Safety In Preclinical Study

  • Avrobio Inc AVRO announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in infantile-onset Pompe disease. 
  • 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). 
  • Also See: AVROBIO Shares Jumps On Encouraging Data From Early-Stage Gene Therapy Study.
  • 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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