Strategies of mRNA- Based Therapeutics to Replace Enzymes for Rare Diseases

Time: 3:00 pm
day: Day Two


  • Exploring mRNA construct requirements to meet therapeutic thresholds for rare diseases
  • Unravelling the challenges expression vs delivery for successful mtranslation of mRNA- based therapeutics
  • How to predict efficacy from pre-clinical models to unleash the therapeutic benefit for patients with rare diseases?