Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper.

ABSTRACT: Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-associated virus (AAV) vector-based gene therapies. Mild to severe adverse events observed in clinical development have been implicated with host immune responses against AAV gene therapies, resulting in comprehensive evaluation of immunogenicity during nonclinical and clinical studies mandated by health authorities. Immunogenicity of AAV gene therapies is complex due to the number of risk factors associated with product components and pre-existing immunity in human subjects. Different clinical mitigation strategies have been employed to alleviate treatment-induced or -boosted immunogenicity in order to achieve desired efficacy, reduce toxicity, or treat more patients who are seropositive to AAV vectors. In t

SUBMITTER: Yang TY 

PROVIDER: S-EPMC9418752 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature