The Food and Drug Administration (FDA) has granted Fast Track designation to GPH101 for the treatment of sickle cell disease.

GPH101 is an investigational gene-edited autologous hematopoietic stem cell therapy (HSCT). It is designed to correct the mutation in the beta-globin gene, thereby reducing sickle hemoglobin (HbS) production and restoring normal adult hemoglobin (HbA) expression.

The Company is evaluating GPH101 in approximately 15 patients with severe sickle cell disease in the open-label, multicenter phase 1/2 CEDAR trial (ClinicalTrials.gov Identifier: NCT04819841). Participants will receive GPH101 via IV infusion following myeloablative conditioning in an autologous HSCT setting.

The primary objective of the study is to evaluate safety; secondary endpoints include an assessment of HbA, HbS and total hemoglobin levels and the effect of treatment on clinical manifestations such as vaso-occlusive crisis and acute chest syndrome.

“The FDA’s decision to grant Fast Track designation to GPH101 for sickle cell disease 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, CEO of Graphite Bio. “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.”

Reference

Graphite Bio announces US FDA Fast Track designation granted to GPH101 for the treatment of sickle cell disease. News release. Graphite Bio, Inc. Accessed May 3, 2022. https://www.businesswire.com/news/home/20220503005073/en/Graphite-Bio-Announces-U.S.-FDA-Fast-Track-Designation-Granted-to-GPH101-for-the-Treatment-of-Sickle-Cell-Disease