Next-Generation Gene Editing

The discovery of Cas9 revolutionized how many diseases can be treated, creating exciting opportunities to target disease drivers in a new way. The ability to specifically change, insert or remove elements of targeted genes, is game-changing, putting potential cures for patients living with genetic disease within reach. While patients are beginning to reap the benefits from first-generation CRISPR-based medicines, the technology has distinct limitations that hinder its widespread application in the development of novel therapeutics. As the field continues to expand, new technologies have enabled a next generation of genomic therapies that hold the potential for more precise gene editing to treat many more diseases and patients.

With a wholly owned suite of nucleases, we are building on the promise of CRISPR-Cas based gene editing by addressing key limitations of Cas9 and expanding access to all sites in the genome. Our diverse gene editing toolbox enables a variety of functions spanning from simple knockdowns of a protein to more sophisticated and precise ways of editing the genome that enable a differentiated approach with broad therapeutic applications.