The CRISPR/Cas9 system can be applied in two ways: in vivo and ex vivo.
in vivo applications
ex vivo applications
A critical element for developing effective CRISPR/Cas9 treatments is creating or enhancing the delivery methods for the technology.
For in vivo applications, Intellia is delivering CRISPR/Cas9 components via our modular lipid nanoparticle (LNP) delivery system. Certain LNPs have demonstrated efficacy, safety and favorable tolerability in animal models and clinical settings. Some LNPs are currently used to deliver therapeutic small interfering RNA (“siRNA”), as well as therapeutic messenger RNA (“mRNA”). Due to our team’s expertise in LNP delivery technology, we expect to be able to readily translate the LNPs that we are using for our preclinical development to clinical development in humans.
For ex vivo opportunities see engineered cell therapy
We are actively investigating complementary delivery methods, including evaluating multiple viral delivery vectors, which are viruses engineered to carry non-viral nucleic acids to patients’ cells, for in vivo delivery as well as several newer technologies for delivery to cells ex vivo, which may provide advantages in delivery efficiency or cell viability.
Intellia’s broad-based intellectual property portfolio encompasses foundational filings on the use of CRISPR/Cas9 systems for genome editing, as well as improvements and modifications of these systems and their components for human therapeutic use; product guides and methods of treatment; mRNA optimization; lipid nanoparticle technologies for delivering protein/nucleic acid complexes and RNA into cells; and cell expansion technology relevant to stem cell-based therapies.