Panel Discussion – Diving into Specific Therapeutic Modalities

Time: 10:40 am
day: Day Two


  • Advantages and disadvantages across small molecules, gene therapy, antisense oligonucleotides, gene replacement, stem cell therapy and hybrid strategies
  • With each modality, what needs to happen before the trial, concurrently with the trial and after?
  • What’s the longevity of these approaches?
  • What are the knock on effects of taking these genes out?
  • What is a good candidate for a gene altering therapy or a disease modifying therapy?
  • What limitations might we face due to public policy?