About Us: Overview
Targeted gene integration to transform lives.
At Graphite Bio, we are rapidly building a next-generation gene editing company that is driven to apply our groundbreaking technology with urgency to advance gene replacement therapies and cures for people living with serious diseases. We stand apart through our ability to harness natural and highly precise cellular DNA repair machinery to achieve high efficiency, targeted gene integration to correct the underlying causes of diseases.
Disrupting the Status Quo in the Gene Editing Field
Our unique approach is designed to overcome past barriers to gene editing, achieving targeted gene integration that builds on first-generation proven CRISPR technology to address the underlying causes of diseases.
Rooted in a Strong Scientific Foundation
Graphite Bio’s approach is based on the extensive work and expertise of our academic co-founders Matthew Porteus, M.D., Ph.D. and Maria Grazia Roncarolo, M.D., Ph.D. at the Center for Definitive and Curative Medicine (CDCM) at Stanford University. Through his work and collaborations, Dr. Porteus led groundbreaking research improving the efficacy and fidelity of CRISPR-Cas9 technologies that are now the gold standard in the field of gene editing.
Our platform technology builds upon the work of academic co-founder and current head of translational science, Dr. Daniel Dever. As a post-doctoral student working closely with Dr. Porteus at Stanford, Dr. Dever conducted gene editing research that demonstrated an increase in DNA integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types. This work forms the foundation of our next-generation platform designed to enable targeted and permanent DNA integration at very high efficiency.
One-Time Treatments, Lifetime of Benefits
Our highly-differentiated, targeted gene integration platform harnesses CRISPR and natural DNA repair processes called homology directed repair (HDR) to precisely replace a damaged portion of a gene or insert new genetic information to treat disease. We are working with urgency to apply the capabilities of our platform to develop potential gene replacement therapies that could offer one-time outpatient cures across a range of genetic and other diseases.