Intellia Flags In Vivo CRISPR Knockout Platform As Key Priority For 2023

06 Jan 2023

• By Ayisha Sharma

After ending 2022 with more than $1bn in cash, the US firm is keen to validate its in vivo approach to gene therapy with investigational new drug applications and pivotal trials lined up for two core rare disease programs.

Blocks with numbers on them being stacked on each other by hand, yellow background — Intellia Is Also Working On A Gene Insertion Technology • Source: Shutterstock

Intellia Therapeutics, Inc. is shifting focus to two in vivo gene editing programs set to enter pivotal development over the next year as the firm prepares for clinical milestones that could validate its ‘knockout’ method and proprietary lipid nanoparticle (LNP) platform.

The Cambridge, MA-based firm said on 5 January it had ended 2022 with $1.3bn in cash and cash equivalents, which...