- Either ImmTOR or an empty nanoparticle (NP) control was coadministered with the AAV8 vector at Day 0 and Day 21
- Either ImmTOR or an empty NP control was coadministered with the AAV8 vector at Day 0. Mice were then challenged with either an AAV8 or an AAV5 vector on Day 21
Mice pretreated with the AAV8 vector in combination with the empty NP control developed a significant immune response when challenged with a redose of AAV8. However, pretreatment with the AAV8 vector in combination with ImmTOR mitigated the formation of anti-AAV8–specific antibodies. The formation of NAbs in mice challenged to a dose of AAV5, regardless of the pretreatment, suggested that targeted immune tolerance, rather than broad immunosuppression, was successfully achieved.
References: 1. Meliani A, Boisgerault F, Hardet R, et al. Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. Nat Commun. 2018;9:4098. 2. Kishimoto TK, Meliani A, Collaud F, et al. Antigen-specific modulation of capsid immunogenicity with tolerogenic nanoparticles results in successful AAV vector readministration. Poster presentation at: 2016 European Society of Gene and Cell Therapy Annual Congress; October 18-21, 2016; Florence, Italy. Poster #047.