Our approach is to first nominate all possible off-target sites using a diverse genomic database representative of your intent-to-treat population to assure adequate breadth of potential risks. Confirmation of off-target edits are conducted in representative in vitro, in vivo, or ex vivo environment. The results would be correlated to pathogenic, oncogenic, and biologic impact information to inform patient therapy decisions.
NoteSeQ is powered by the ONE-seq assay technology which leverages customizable, high-throughput DNA synthesis technology to profile off-target sites. Along with an accompanying bioinformatics platform and a non-homology based orthogonal assay the NoteSeQ platform represents a population-based, scalable, and editor-agnostic suite of tools and analyses that are best in class for nominating potential off-target sites.
ScopeSeQ is our confirmation platform that includes tools and analyses to comprehensively confirm candidate off-target edits for a given individual patient genome or tissue, a targeted therapeutic population with unique population genetics, or an allogeneic cell line.