Graphite Bio announced the dosing of the first patient with GPH101, now known as nulabeglogene autogedtemcel (nula-cel), in their phase 1/2 CEDAR trial for patients with sickle cell disease (SCD).
The announcement, reported by news release, noted that the therapy is designed to definitively cure the disease by correcting the causative genetic mutation in SCD.
“For decades, the goal of gene editing has been to precisely correct genetic mutations that cause disease. Today, we took an important step toward achieving that goal by dosing our first patient with nula-cel, the first investigational therapy designed to correct a mutated gene to normal,” Josh Lehrer, MD, MPhil, chief executive officer of Graphite Bio, said.
“This first use of high-efficiency precision DNA repair to correct a genetic mutation is an important milestone not only for our company but also for the gene editing field and, hopefully, for the sickle cell community.”
Read more about SCD experimental therapies
Nula-cel is a next-generation gene-editing autologous hematopoietic stem cell therapy. It was formerly known as GPH101, and it was granted Orphan Drug and Fast Track designations by the US Food and Drug Administration (FDA) for the treatment of SCD.
Nula-cel is being tested in CEDAR, an open-label, single-dose study. The CEDAR study aims to assess the safety, efficacy, and pharmacodynamics of the compound in 15 individuals with severe SCD. Nula-cel aims to efficiently and accurately correct the beta-globin gene mutation, thereby reducing the production of sickle hemoglobin and restoring proper adult hemoglobin expression. If successful, it represents a potentially definitive cure for the disease.
Graphite Bio doses first patient with investigational gene editing therapy GPH101 for sickle cell disease. News release. Graphite Bio, Inc; August 11, 2022.