As CRISPR-based therapies advance rapidly, ensuring off-target safety is critical, especially when timelines are short and clinical stakes are high.
This webinar introduces a comprehensive, scalable framework for off-target risk assessment that integrates orthogonal nomination strategies, including cell‑based (UNCOVERseq), in vitro (CHANGE-seq and ONE-seq), and in silico approaches. Designed to support rapid decision-making, this framework also helps researchers and developers prioritize and confirm off-target sites using targeted deep sequencing with our rhAmpSeq™ CRISPR Analysis System.
Case study overview:
We'll walk you through how this approach can be applied across a range of gene editing programs, with a case study highlighting its use in a time-sensitive pediatric setting involving a novel base editor for a severe urea cycle disorder with results published in the New England Journal of Medicine.
