TFRC-Targeted GAA Delivered as Gene Therapy Treats CNS & Muscle in Pompe Disease Model Mice

Time: 3:40 pm
day: Day One


  • Design and selection of anti-TFRC antibody fused to GAA
  • Delivery of anti-TFRC:GAA as a liver depot gene therapy in Pompe disease Gaa-/- mice results in targeted uptake of GAA protein in need-to-treat tissues, including key cell types in the brain
  • Anti-TFRC:GAA rescues glycogen storage in CNS, heart, and skeletal muscle in Gaa-/- mice