GPH102

GPH102 is an experimental ex vivo gene editing therapy developed by Graphite Bio for the treatment of beta-thalassemia. Utilizing the company's proprietary UltraHDR™ gene editing platform, GPH102 employs a CRISPR-based gene replacement mechanism to replace the mutated beta-globin (HBB) gene with a functional copy in a patient's own hematopoietic stem and progenitor cells (HSPCs). This approach is intended to restore the production of adult hemoglobin (HbA) to levels similar to those in healthy individuals. The treatment process involves the ex vivo modification of autologous HSPCs followed by their transplantation back into the patient after a conditioning regimen. In early 2023, Graphite Bio announced it would discontinue the development of its internal programs, including GPH102, as part of a strategic restructuring and subsequent merger with LENZ Therapeutics.