GPH301

GPH301 is a next-generation, ex vivo gene-edited autologous hematopoietic stem cell therapy developed by Graphite Bio (now LENZ Therapeutics) for the treatment of Gaucher disease, particularly Types 1 and 3. Using a CRISPR-based targeted gene insertion approach, GPH301 is designed to insert a functional copy of the GBA1 gene encoding glucocerebrosidase into the CCR5 “safe harbor” locus of a patient’s own hematopoietic stem cells, enabling stable, long-term systemic production of the missing lysosomal enzyme as a potentially one-time curative alternative to chronic enzyme replacement therapy. The program remained in preclinical/IND-enabling development and has since been terminated along with Graphite Bio’s broader CRISPR HSC platform programs.[1][2][4][5][9][11][13]