Yang, Tong-yuan, Braun, Manuela, Wibke, Lembke, McBlane, Fraser, Kamerud, John, DeWall, Stephen, Tarcsa, Edit, Fang, Xiaodong, Hofer, Lena, Kavita, Uma, Upreti, Vijay V., Gupta, Swati, Loo, LiNa, Johnson, Alison J., Jawa, Vibha, Chandode, Rakesh Kantilal, Vinzing, Maya and Xia, Cindy Q. (2022) Immunogenicity Assessment of AAV-based Gene Therapies: An IQ Consortium Industry White Paper. Molecular therapy - methods and clinical development, 26. pp. 471-494. ISSN 2329-0501

Abstract

Immunogenicity is critical to AAV-based gene therapy during discovery and development. The cross industry working groups organized by International Consortium for Innovation and Quality in Pharmaceutical Development (IQ) have focused on various aspects of critical questions related to AAV-based gene therapy. This white paper summarizes the immunogenicity potential and risks associated with recombinant AAV-based GTs based on current clinical experiences and provides a framework for bioanalytical approaches related to the immunogenicity assessment of rAAV-based GTs. The paper provides a comprehensive overview of immunogenicity risks associated with rAAV GTs, as well as describes the methodologies for monitoring pre-existing and treatment boosted/or induced immune responses (humoral vs cellular). In addition, we discuss current clinical mitigation strategies deployed to reduce immunogenicity upon administration of rAAV-based GT

Item Type:	Article
Date Deposited:	30 Nov 2022 00:45
Last Modified:	30 Nov 2022 00:46
URI:	https://oak.novartis.com/id/eprint/46549