ScienceDirect ® is a registered trademark of Elsevier B.V. ScienceDirect ® is a registered trademark of Elsevier B.V. Vol. Though the genetic ciphering remains largely unchanged through generations, some genes go awry due to mutations, and disruptions or deletions (5). Gene Therapy Gathers Momentum Those who have followed the gene-therapy field over the decades may be weary of forward-looking positive statements. We use cookies to help provide and enhance our service and tailor content and ads. The approved gene therapy drugs worldwide: from 1998 to 2019. Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. "It took a generation for gene therapy to become a reality. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris. Until August 2019, 22 gene medicines had been approved by the drug regulatory agencies from various countries, but there were few relevant reviews of combing these drugs systematically. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris. Until August 2019, 22 gene medicines had been approved by the drug regulatory agencies from various countries, but there were few relevant reviews of combing these drugs systematically. With the improvement of gene vectors, the rise of chimeric antigen receptor T cell immunotherapy and breakthroughs in the genome editing technology, gene therapy had once again returned to the central stage of disease treatment. ARM Annual Report & Sector Year in Review: 2019 The 2019 ARM Annual Report provides an overview of the organizations events, publications, and policy initiatives over the last year, with commentary from ARM members. © 2020 Elsevier Inc. All rights reserved. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed vectors. Now, it's only going to go faster." 70:273-288 (Volume publication date January 2019) First published as a Review in Advance on November 26, 2018 ... this review focuses on the clinical research that has forged the gene therapy field as it currently stands. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. Copyright © 2020 Elsevier B.V. or its licensors or contributors. Gene therapy had gradually been accepted by the government and the public since 1980s, and have become a new and important alternative to existing treatments for human diseases in the past few years. Now, it's only going to go faster." "It took a generation for gene therapy to become a reality. Gene Therapy N Engl J Med. Achieved or exceeded all MDUFA review performance goals for FY 2019. Keywords. These underlying and inevitable genetic changes translate into altered p… Global Cell Therapy and Gene Therapy Markets, 2019-2020 & 2030 - Dermatology, Cardiovascular and Blood Disorders, Oncology, Ophthalmic Conditions, Musculoskeletal, Market Review & Market Participants These authors contributed equally to this work. 1 and the details of approved gene therapy drugs were exhibited in Table 1, respectively.This review concluded the approved gene therapy drugs by the drug regulatory agencies of various countries or regions from August 1998 to August 2019, and addressed opportunities and challenges for developing gene therapy drugs. gene therapy, lentiviral vectors, adeno-associated viral vectors, immunotherapy. However, over the past 3 years, six gene-therapy … By continuing you agree to the use of cookies. It had brought new choices to clinical therapy of diseases such as tumors and genetic diseases, and had changed the status quo of treatment for monogenic disorders and diffuse large B-cell lymphoma. https://doi.org/10.1016/j.biotechadv.2019.107502. Gene Therapy. Gene Therapy. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910. The human genome contains ~25,000 genes that encode a wide variety of proteins colloquially called the building blocks and workhorses of the cell to drive every biological process necessary for life and death (1–4). The timeline of approved gene therapy drugs was shown in Fig. Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Therefore, gene therapy drugs, with safe vectors and advanced biotechnologies, would play a greater role in the prevention and treatment of human diseases in future.