Approach to mitigate on-target toxicity using knock out animals and in vitro assay system at exploratory phase

Abstract

To mitigate on-target toxicity at exploratory phase is a difficult challenge. Exploratory toxicology study using knock-out animals for the target genes is widely used to determine if the toxic finding due to the candidate is on-target or not. In case, on-target toxicity is caused by the candidate targeted for unknown gene, alternative approaches are necessary to identify whether this on-target toxicity can be mitigated. In this presentation, our approaches to mitigate on-target toxicity using knock out animals and in vitro assay system at exploratory phase will be introduced and discussed.