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.

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.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER