Armed with pub­lished an­i­mal da­ta, gene-edit­ing pi­o­neer In­tel­lia maps the road to an IND

One of the few pi­o­neers in the bur­geon­ing gene edit­ing field, In­tel­lia $NT­LA has di­rect­ed at­ten­tion to its IND-en­abling an­i­mal da­ta for some time. Now, it’s come out with a pub­lished sci­en­tif­ic study — com­plete with a few “un­der the hood” high­lights — to back the hype up.

The study, pub­lished in Cell Re­ports Tues­day, for­mal­ly sum­ma­rizes what In­tel­lia has been tout­ing since last March: the lipid nanopar­ti­cle tech­nol­o­gy it’s used to de­liv­er CRISPR/Cas9 gene edit­ing has proven to es­sen­tial­ly elim­i­nate all transthyretin (TTR) pro­teins in the liv­er of mice. The ef­fects of one dose last­ed for at least 12 months, sug­gest­ing that the gene edit­ing hap­pened not on­ly in nor­mal cells but the stem cell pop­u­la­tion of the he­pa­to­cytes.

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