Our Science: Pipeline

Graphite Bio brings an ambitious vision to advance gene editing from the bench to the bedside – transforming our founders’ ground-breaking discoveries into therapies that are intended to make a meaningful difference in patients’ lives. We are uniting advancements in hematopoietic stem cell (HSC) transplantation and gene editing with the goal of ultimately creating next-generation therapies with broad potential.

Our Pipeline

Gene correction: GPH101 for sickle cell disease

Graphite Bio’s first clinical candidate, GPH101, is an investigational next-generation gene-edited autologous HSC product candidate that is currently in development for sickle cell disease (SCD). SCD is a group of rare inherited blood disorders and among the most prevalent monogenic diseases. It is estimated that there are 100,000 people living with SCD in the United States and millions worldwide. With GPH101, Graphite Bio aims to directly correct the underlying mutation that causes SCD to decrease the production of sickle hemoglobin and restore the expression of normal adult hemoglobin, thereby potentially curing the disease.

GPH101 is being studied in the CEDAR trial, a Phase 1/2 multi-center, open-label clinical trial designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.

Learn more about the Phase 1/2 CEDAR clinical trial here.

Gene replacement: GPH102 for beta-thalassemia

GPH102 is Graphite Bio’s research program for the treatment of beta-thalassemia, a genetic blood disorder that reduces the production of hemoglobin, resulting in severe anemia and in severe cases transfusion dependency. It is one of the most common autosomal recessive disorders in the world, with approximately 68,000 people born with it worldwide each year. GPH102 uses the company’s gene replacement technology to replace the entire mutated beta-globin (HBB) gene and restore adult hemoglobin expression to levels similar to individuals who do not have disease or have beta-thalassemia trait.

Discovery research: Non-genotoxic conditioning regimens

Graphite Bio has ongoing discovery research efforts to develop potential best-in-class non-genotoxic HSC targeted conditioning (NGTC) regimens, which could significantly broaden the number of diseases and patients who can be treated with the company’s precision engineered, one-time treatments and cures. The company also intends to leverage industry advances to accelerate its efforts in this area.