Graphite Bio reports GPH102 preclinical HDR rates up to 40% in sickle cell HSPCs; IND planned mid-2024

LENZ Therapeutics, Inc.

GPH102 gene replacement achieved homology directed repair (HDR) rates up to 40% in sickle cell HSPCs and restored adult hemoglobin (HbA) expression.
Company plans to submit an IND for GPH102 in beta-thalassemia by mid-2024, pending regulatory feedback.
An encore trial-in-progress poster for GPH101 Phase 1/2 CEDAR trial in sickle cell disease was also presented at ASGCT.
GPH102 uses UltraHDR platform to replace mutated beta-globin gene, targeting >300 mutations that cause beta-thalassemia.

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