LV-HSC Platelet-Derived Factor VIII “Pleightlet™” for Severe Hemophilia A with Inhibitors

Time: 4:30 pm
day: Day One


  • Summary of HSC gene therapy strategy for using platelet FVIII for hemophilia A
  • Seminal pre-clinical and validation studies – in support of clinical efficacy
  • Current status of the FDA approved phase I clinical trial