GRPH - Enrollment underway in Graphite Bio's mid-stage GPH101 trial in sickle cell disease

Graphite Bio (NASDAQ:GRPH) announces that the first patient has been enrolled in its Phase 1/2 clinical trial of GPH101, an investigational gene-edited autologous hematopoietic stem cell therapy designed to correct the genetic mutation that causes sickle cell disease (SCD). The company expects to treat the first patient in H1 2022, with initial proof-of-concept data anticipated by end of 2022. The Phase 1/2 clinical trial of GPH101 is designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics of GPH101 in patients with severe SCD. The trial will enroll approx. 15 adult and adolescent participants. SCD is a serious, life-threatening inherited blood disorder, caused by a single mutation in the beta-globin gene that leads red blood cells to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span.

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Enrollment underway in Graphite Bio's mid-stage GPH101 trial in sickle cell disease