Nulabeglogene autogedtemcel

Nulabeglogene autogedtemcel (also known as nula-cel or GPH101) is an investigational, gene-edited, autologous hematopoietic stem cell therapy developed to treat sickle cell disease (SCD). The therapy uses CRISPR/Cas9-based gene editing to precisely correct the pathogenic mutation in the beta-globin gene responsible for SCD. By directly repairing this mutation in a patient’s own CD34+ hematopoietic stem cells, the treatment aims to suppress production of sickle hemoglobin (HbS) and restore healthy adult hemoglobin (HbA), potentially offering a curative approach. The technology originated at Stanford University and was initially developed by Graphite Bio before being acquired by Kamau Therapeutics. Nulabeglogene autogedtemcel received Orphan Drug and Fast Track designations from the FDA for SCD but its development was paused following adverse events in clinical trials[1][3][4][5][6][7].

Other names

Autologous CD34+ Hematopoietic Stem Cells - LENZ Therapeutics