Key information

🔎 Fund Overview

Graphite Bio, Inc. is a clinical-stage gene editing company harnessing targeted gene integration to develop a class of therapies to cure a range of serious and life-threatening diseases. The Company has a gene editing platform that is designed to correct mutations, replace entire disease-causing genes with normal genes, or insert new genes into predetermined, safe locations. Its research program, GPH102 for the treatment of beta-thalassemia, leverages its gene replacement platform technology by replacing one or both copies of the mutated beta-globin (HBB) gene through HDR to restore HgbA expression to levels. GPH201 is an investigational treatment for X-linked severe combined immunodeficiency syndrome (XSCID), a rare, life-threatening disease where multiple mutations in a single gene (IL2RG) prevent normal immune system function. GPH301 is for the treatment of Gaucher disease. Its pipeline program also includes therapeutic protein production and non-genotoxic conditioning (NGTC).