Clinical Safety Monitoring for AAV Gene Therapies 101

Time: 8:30 am
day: Pre-Conference Day


A major objective of early-phase clinical trials is the safety evaluation of a drug candidate. With gene therapies, a complex mixture of difficult-to-predict immediate hypersensitivity and delayed immune reactions can occurs from anywhere between minutes and months of administration. As such, it is vitally important to outline the immune monitoring strategies as part of your clinical safety monitoring strategy ahead of entering the clinic.

Attend this workshop to learn about:

  • Key considerations for measuring the cellular and humoral immune responses to both the vector and transgene
  • How to find out, control, and measure critical assay variables for the cellular response
  • New immune monitoring strategies in the clinic and the rationale behind their selection
  • Exploring some key challenges and limitations (e.g., reproducibility, clinical translatability) of these common assays
  • Should drug sponsors be testing for adaptive cellular immune responses as part of their long-term follow-up plan?
  • Utilising new FDA Type D meetings to help find resolution to safety challenges sooner