Graphite Bio announced that their investigational sickle cell disease (SCD) treatment, GPH101, received Fast Track Designation from the US Food and Drug Administration (FDA). The therapy is based on high-efficiency precision gene repair and has the potential to treat or cure SCD by correcting the genetic mutation that causes the disease.

“The FDA’s decision to grant Fast Track Designation to GPH101 for [SCD] signifies the need for novel medicines for this serious genetic disease and supports the ongoing development of our unique gene correction approach that we believe could offer a definitive cure for sickle cell patients,” said Josh Lehrer, MD, MPhil, chief executive officer of Graphite Bio.

“This designation has the potential to accelerate the development of GPH101, which we are advancing with the goal of precisely and efficiently correcting the genetic mutation that is the underlying cause of [SCD]. We continue to enroll patients in our Phase 1/2 CEDAR trial and expect to dose our first patient later this year, with initial proof-of-concept data anticipated next year.”

Read more about SCD therapies

GPH101 is an investigational next-generation gene-editing approach that uses autologous hematopoietic stem cells to accurately and efficiently correct the beta-globin gene mutation in patients with SCD, which would reduce the production of sickle hemoglobin and increase the expression of normal adult hemoglobin. Graphite Bio is evaluating the therapy as part of the Gene Correction in Autologous CD34+ Hematopoietic Stem Cells to Treat Severe Sickle Cell Disease trial, an open-label, multicenter phase 1/2 trial in patients with severe SCD.

The primary endpoints of the trial are the success of the engraftment, rates of gene correction, total hemoglobin, and other parameters. The trial is currently enrolling patients and the first dose is expected to be administered later in 2022, with preliminary results expected in 2023.


Graphite Bio announces U.S. FDA fast track designation granted to GPH101 for the treatment of sickle cell disease. News release. Graphite Bio; May 3, 2022.