Senior Director Translational Bioanalytical Oversight Lilly Havertown
Adeno-associated viral (AAV) vectors have emerged as a delivery tool for treating various diseases. To date, seven AAV-based gene therapies (Beqvez™, Elevidys®, Hemgenix®, Kebilidi/Upstaza™, Luxturna®, Roctavian®, and Zolgensma®) have been approved, and there are hundreds of clinical trials to evaluate AAV vectors in the central nervous, ocular, auditory, cardiovascular, and immune systems via local or systemic routes of administration. In AAV -based gene therapy, shedding refers to the release of vector into bodily fluids or excreta of treated subjects. It is a key safety assessment in clinical trials, as persistent shedding can indicate the presence of unwanted replication of the vector, and the vectors may go beyond the treated individual. To gain insight on vector shedding study strategies, we reviewed information from clinical trials in various databases. In this presentation, we share our learnings and discuss the shedding study designs with different routes of administration and for diverse indications.
Learning Objectives:
Understanding AAV based gene therapy and the importance of conducting vector shedding study
Obtain an overview on vector shedding study designs in various clinical trials, and understand how different clinical trial parameters impact vector shedding study design.
Discuss best practice for future shedding study design
