In­tel­lia fine tunes its CRISPR/Cas9 tech ahead of hu­man stud­ies

One of the hand­ful of gene ther­a­py star­tups has been fine tun­ing its CRISPR/Cas9 tech in the lead up to tak­ing it in­to hu­mans.

Build­ing on pre­clin­i­cal work pub­lished last sum­mer, in­ves­ti­ga­tors at Cam­bridge, MA-based In­tel­lia $NT­LA say they’ve sub­stan­tial­ly im­proved on the ef­fi­cien­cy of their gene edit­ing work, gain­ing a 97% re­duc­tion in serum transthyretin (TTR) pro­tein dri­ven by 70% gene edit­ing ef­fi­cien­cy work­ing in mouse liv­ers. That was with a sin­gle IV in­fu­sion us­ing its lipid nanopar­ti­cle de­liv­ery tech. And Cas9 mR­NA and guide RNA were un­de­tectable in the liv­er 72 hours af­ter the in­fu­sion.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.