GSK submits MAA for ADA-SCID gene therapy

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) submitted an MAA to EMA for autologous gene therapy GSK2696273 to treat severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency (ADA-SCID).

The ultra-rare disease is caused by inheritance of two mutant copies of the gene encoding ADA and results in an inability to make lymphocytes. GSK2696273 uses a retroviral vector to deliver a functional ADA gene into a patient's own hematopoietic stem cells. The cells are harvested from bone marrow, transfected ex vivo and injected back into the patient. ...