Next-Generation Gene Editing

The discovery of Cas9 revolutionized how genetic disease can be treated, creating new and exciting opportunities. The ability to cut DNA and change, insert or remove elements of targeted genes was game-changing, putting potential cures for patients with genetic disease within reach. While patients may soon reap the benefits from first-generation CRISPR-based medicines, the field continues to expand, unearthing a next-generation of genomic therapies that hold the potential for more precise gene editing to treat many more diseases and many more patients.

With a suite of nucleases with the PAM diversity to target more than 93% of the human genome, our toolbox enables a variety of functions, spanning from simple knockdowns of a protein to more sophisticated and precise ways of editing the genome. We have chosen to focus our efforts on four primary platforms for editing DNA, each of which contain unique properties that enable a differentiated approach that can be used for many diseases.