San Francisco-based Graphite Bio announced that it has received Fast Track Designation from the U.S. Food and Drug Administration for its investigational therapy GPH101, which is designed to genetically treat sickle cell disease (SCD). Sickle cell disease has a high unmet need, and since several other companies have recently hit roadblocks with their candidates, the Fast Track Designation is a huge win for both Graphite Bio and patients.

GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy. Graphite Bio, which specializes in precision gene editing, developed GHP101 to treat sickle cell disease with a two-pronged approach. GHP101 attempts to genetically correct a mutation in the beta-globin gene in order to both decrease production of sickle hemoglobin and restore adult hemoglobin expression. By resolving these two issues, GHP101 could potentially cure the disease. The treatment received FDA investigational new drug (IND) clearance in December 2020. It also received Orphan Drug Designation. Since then, Graphite Bio has been evaluating the efficacy of GHP101 in clinical trials. Its CEDAR study, a Phase I/II clinical trial, began enrolling patients in November 2021. Read more here.