![]() Ikeda) Cincinnati Children's Hospital Medical Center, Cincinnati (L.R.) Indiana University School of Medicine, Indianapolis (K.C.) Duke University, Durham, NC (R.P., R.H.B., M.H.) Division of Immunology and Allergy, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland (F.C.) and GeneWerk (M.Z., R.H., I.L., M.S.) and the German Cancer Research Center and the National Center for Tumor Diseases (M.Z., M.S.) - all in Heidelberg, Germany. Snell, D.L.-R., K.F.B., K.C.G., C.R., N.I., S.A., H.R., C.U., A.J.T., H.B.G.), and Orchard Therapeutics (Europe) (D.A.C.-S., S.A., F.L., M.K., A.S., H.B.G.) - all in London the National Institute of Allergy and Infectious Diseases (H.L.M.) and the National Human Genome Research Institute (E.G., R.S., F.C.), National Institutes of Health, Bethesda, MD Cure 4 The Kids Foundation, Las Vegas (A. Icreverzi, P.B., B.C.F., R.P.H., M.C., A.Y., K.M.C., C.E.C., R.Z.), Pediatrics (D.B.K., T.B.M., S.D.O., S.S.), and Pathology and Laboratory Medicine (G.M.C.) and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (D.B.K., G.M.C.), University of California, Los Angeles (UCLA), and the Department of Pharmaceutical Services, Ronald Reagan UCLA Medical Center (J.T.), Los Angeles, and Stanford School of Medicine, Palo Alto (A.J.S.) - all in California University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust (C.B., J.X.-B., V.T., K. 1 From the Departments of Microbiology, Immunology, and Molecular Genetics (D.B.K., K.L.S., D.A.C.-S., D.T., A.D., A.Science 270: 475–480īordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P, Mazzolari E, Maggioni D, Rossi C, Servida P et al (1995) Gene therapy in peripheral blood lymphocytes and bone marrow for ADA‐ immunodeficient patients. N Engl J Med 360: 447–458īlaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P et al (1995) T lymphocyte‐directed gene therapy for ADA‐ SCID: initial trial results after 4 years. J Clin Invest 117: 2233–2240Īiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I et al (2009) Gene therapy for immunodeficiency due to adenosine deaminase deficiency. Science 296: 2410–2413Īiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M et al (2007) Multilineage hematopoietic reconstitution without clonal selection in ADA‐SCID patients treated with stem cell gene therapy. Strimvelis™ consists of a single infusion of autologous gene‐corrected HSC and is prepared from the patient's own bone marrow ( BM) HSCs, which are genetically modified using a gamma‐retroviral vector to insert a functional copy of the ADA gene.Īiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, Morecki S, Andolfi G, Tabucchi A, Carlucci F et al (2002) Correction of ADA‐SCID by stem cell gene therapy combined with nonmyeloablative conditioning. This ATMP is the first ex vivo stem cell gene therapy to receive regulatory approval anywhere in the world. The new medicine, named Strimvelis™, is an advanced therapy medicinal product ( ATMP) (Salmikangas et al, 2015) originally developed by the San Raffaele Telethon Institute for Gene Therapy ( SR‐Tiget), a joint venture between Telethon Foundation and San Raffaele Scientific Institute. In 2016, the European Commission granted market approval to GlaxoSmithKline ( GSK) for ex vivo hematopoietic stem cell ( HSC) gene therapy for the treatment of adenosine deaminase ( ADA)‐deficient severe combined immunodeficiency ( SCID), a very rare congenital disorder of the immune system. ![]() Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases.
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