Lessons Learned From Potency Assays For Gene Therapy Drug Products

Time: 1:30 pm
day: Track A - Day 1 PM


  • Quantitative potency assays were developed to demonstrate correction of b-thalassemia and sickle cell disease properties in an in-vitro cell culture system.
  • Considerations for assay development, qualification results, and redundancy to transduction efficiency methods will be discussed.