AAVrh10-based Gene Therapy for the Treatment of Frontotemporal Dementia Caused by GRN Mutations

Time: 5:00 pm
day: Day One


  • Dominant mutations in granulin (GRN) gene accounts for up to 10% of frontotemporal dementia (FTD) and results in > 50% reduction of progranulin (PGRN) expression in patients
  • Adeno-associated virus (AAV) gene therapy to booster the levels of progranulin may represent a feasible strategy to treat FTD-GRN
  • AGTC-601 is an AAV gene therapy to supplement progranulin expression in brain to treat FTD-GRN and shows promising efficacy and safety profile