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. The Company is uniting advancements in hematopoietic stem cell (HSC) transplantation and gene editing to ultimately create next-generation therapies with broad potential.
GPH101: Distinct approach in sickle cell disease
Graphite Bio’s first clinical candidate, GPH101, is a best-in-disease Phase 1/2 program for sickle cell disease (SCD).
GPH101 is a first-in-industry approach to correct sickle mutation and restore normal adult hemoglobin by directly targeting the ß globin gene. GPH101is anticipated to enter clinical testing in 2021 in the CEDAR trial. CEDAR is a Phase 1/2, multi-center, open-label clinical study designed to evaluate the safety, preliminary efficacy and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD.
GPH201: Novel gene replacement therapy for severe combined immune deficiency
Graphite Bio’s second lead gene replacement therapy candidate is GPH201, a first-in-human investigational hematopoietic stem cell treatment that will be evaluated as a potentially curative therapy for patients with severe combined immune deficiency (SCID) with IL2RG deficiency, known as x-linked SCID (XSCID).
GPH201 utilizes Graphite Bio’s precise and efficient targeted gene integration platform to directly replace the defective IL2RG gene, maintain normal IL2RG regulation and expression, and ultimately lead to the production of fully functional adaptive immune cells.