The Pharmacology of Gene and Cell Therapy

• Published in: Molecular therapy. Methods & clinical development Vol. 8; no. C; pp. 181 - 182
• Main Authors: Conlon, Thomas J., Mavilio, Fulvio
• Format: Journal Article
• Language: English
• Published: United States Elsevier Limited 16.03.2018; American Society of Gene & Cell Therapy; Elsevier
• Subjects: Autografts; Biological products; Cancer; Clinical trials; Deoxyribonucleic acid; DNA; Drug dosages; Federal regulation; Gene expression; Gene therapy; Genetic engineering; Genomes; Hematopoietic stem cells; HIV; Human immunodeficiency virus; Immunotherapy; Lipase; Lipoprotein lipase; Lymphocytes; Lymphocytes T; Manufacturing; Oncolysis; Pharmacology; Retina; Retinal degeneration; Severe combined immunodeficiency; Stem cells; Vectors (Biology); Viruses; United States > US; Europe
• ISSN: 2329-0501; 2329-0501
• DOI: 10.1016/j.omtm.2017.12.007

Glybera is a recombinant adeno-associated virus (AAV) vector designed for gene therapy of lipoprotein lipase deficiency,1 while Strimvelis is a genetically modified hematopoietic stem cell preparation for the treatment of severe combined immunodeficiency.2 The US Food and Drug Administration (FDA) recently filled the gap for the US market by approving in rapid succession Ymlygic, a genetically modified oncolytic herpes virus, Kymriah and Yeskarta, two forms of genetically manipulated autologous T cells for immunotherapy of cancer, and Luxturna, a recombinant AAV vector for gene therapy of a rare form of retinal dystrophy. Regulatory agencies around the world are trying to cope by providing guidelines and regulations intended to enable development and marketing of gene therapy in a safe, sensible fashion to avoid unnecessary delays in making innovative products available to patients.3 This issue of Molecular Therapy – Methods & Clinical Development provides an update on some outstanding issues in the pharmacology of gene therapy products. Poletti and Mavilio7 describe the molecular basis of target site selection by retroviruses and retroviral vectors, a knowledge derived from over 15 years of research on the biology of HIV and the severe adverse events observed in gene therapy clinical trials.