Analytical Development Challenges with AAV Treatments for Ultra-rare Diseases

Time: 11:30 am
day: Track A - Day 2 AM


  • Developing Analytical specifications for clinical and commercial lots is challenging due to the minimum number of lots that may be produced during an ultra-rare disease program
  • The function of the defective gene in ultra-rare diseases may not be known
  • Potency assays therefore may be based on a matrix approach