base-edited autologous hematopoietic stem and progenitor cells

This investigational ex vivo gene therapy consists of autologous hematopoietic stem and progenitor cells (HSPCs) that have been genetically modified using adenine base editors (specifically ABE8e-SpRY). Developed by the National Institute of Allergy and Infectious Diseases (NIAID) in collaboration with Massachusetts General Hospital, the therapy is designed to treat X-linked chronic granulomatous disease (X-CGD) by correcting disease-causing A>G mutations in the CYBB gene. By repairing the genetic defect in the patient's own stem cells, the therapy aims to restore the production of functional NADPH oxidase in phagocytes, thereby reducing the high susceptibility to life-threatening infections characteristic of X-CGD. The edited cells are re-infused into the patient following a conditioning regimen. It is currently being evaluated in a Phase 1/2 clinical trial (NCT06325709).

Other names

Base-edited hematopoietic stem and progenitor cellsBase-edited autologous CD34+ HSPCsABE8e-SpRY edited HSPCsABE-8e-SpRY edited HSPCsABE 8e-SpRY edited HSPCs