GSK wins European regulator nod for new gene therapy

GlaxoSmithKline ($GSK) has won a key recommendation from the European regulator EMA today for Strimvelis (GSK2696273)--a groundbreaking new gene therapy for children living with the incredibly rare and fatal condition, severe combined immunodeficiency due to adenosine deaminase deficiency. The drug works by using a viral vector to insert working copies of the ADA gene into stem cells extracted from the bone marrow of patients. The cells are then reintroduced to the patient, who can expect to start making the gene on their own, repairing their immune system. The treatment, which Glaxo is making alongside Italian partner San Raffaele Telethon Institute for Gene Therapy and with help from Milanese biotech MolMed, should receive a final European Commission approval in around three months. Release