The Power of Naturally Occurring AAV Capsids for the Development of Gene Transfer & Gene Editing Therapies

Time: 10:00 am
day: Post-Conference Delivery Day


  • Diving into the structure/function relationship of unique AAVHSCs, a panel of naturally occurring AAV capsids isolated from human hematopoietic stem cells
  • Highlighting AAVHSC16, an AAVHSC capsid which displays significantly lower liver tropism compared to other Clade F capsids while retaining robust tropism to a wide variety of peripheral and CNS tissues
  • AAVHSC16 does not lead to the induction of liver transaminases after high dose intravenous administration in NHPs, making AAVHSC16’s safety profile attractive for the development of gene therapies