Graphite Bio announced the enrollment of the first patient in their phase 1/2 clinical trial of GPH101 (NCT04819841) for the treatment of sickle cell disease (SCD). The company expects to treat this patient in the first half of 2022 and hopes to get initial proof-of-concept data by the end of 2022.

GPH101 is an investigational therapy comprised of gene-edited CD34+ hematopoietic stem cells (HSCs). The HSCs have been edited using CRISPR-Cas9 technology. The treatment is designed to correct the mutation in the beta-globin gene responsible for causing SCD. 

“GPH101 is the first investigational therapy to enter clinical development that uses our next-generation gene-editing platform technology to directly correct the mutation in the beta-globin gene that causes sickle cell disease,” Josh Lehrer, MPhil, MD, chief executive officer at Graphite Bio said.

“Using our gene correction approach, we have demonstrated in preclinical studies an ability to decrease the production of harmful sickle hemoglobin and restore the expression of normal adult hemoglobin,” Dr. Lehrer continued. “This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease.”

Read more about SCD experimental therapies

The patient is enrolled as part of the open-label, multicenter CEDAR phase 1/2 clinical trial. The trial aims to recruit 15 patients with SCD who are aged 12 to 40 years to receive an intravenous infusion of GPH101 following a myeloablative conditioning regimen.

The trial will evaluate the safety, engraftment success, gene correction rates, total hemoglobin levels, pharmacodynamics, and other clinical endpoints. The trial is currently recruiting patients at trial sites in California and Missouri but may add up to 3 additional sites.

The company will present more information about the clinical trial in a poster at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition on December 11, 2021.

“We are thrilled that our first patient is now enrolled in our CEDAR clinical trial, and we look forward to evaluating GPH101’s potential as we continue to advance its development with urgency in hopes of delivering a curative therapy to the sickle cell community,” Dr. Lehrer said.


Graphite Bio enrolls first patient in phase 1/2 clinical trial of GPH101 for sickle cell disease. News Release. Graphite Bio; November 17, 2021.

Gene correction in autologous CD34+ hematopoietic stem cells (HbS to HbA) to treat severe sickle cell disease (CEDAR). US National Library of Medicine. Accessed November 26, 2021.