Multiple Genome Editing Approaches

Genetically driven diseases caused by mutations come in many different forms, ranging from those that reduce or eliminate gene function, to those that direct the production of pernicious or damaging proteins. Intellia is applying and optimizing its CRISPR/Cas9 genome editing platform to address each of these types of mutations.

In vivo: CRISPR is the therapy

Our systemic lipid nanoparticle (LNP)-based delivery system has unlocked treatment of genetic diseases to both selectively knock out disease-causing genes and restore necessary genetic functions by targeted insertion.

Ex vivo: CRISPR creates the therapy

We are focused on engineering T cellcell therapies to provide them with particular enhanced attributes that may enable them to more effectively treat oncological and immunological diseases. Our approach is designed to improve safety and efficacy by engineering cell therapies that are more precise, potent and persistent.