Plenary Lecture II: Realizing the Promise of In Vivo CRISPR Therapeutics

Intellia is leveraging CRISPR/Cas9 technologies for therapeutic genome editing. Our systemic lipid nanoparticle (LNPs) delivers a messenger RNA that encodes the Cas editing enzyme and a guide RNA that targets the desired genomic sequence to be edited. LNPs are given IV for in vivo genome editing, as well as used ex vivo, for engineering cell therapies. Preclinical and clinical data of pipeline programs will be discussed.