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Amit C. Nathwani; Gene therapy for hemophilia. © 2020 Elsevier Inc. All rights reserved. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed vectors. 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. 2. Since the approval of the first therapy, Genedicine® in 2003, the gene therapy domain has evolved significantly. The hemophilias are ideally suited for gene therapy because a small increment in blood factor levels (≥5% of normal) is associated with significant amelioration of bleeding phenotype in severely affected patients. 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. 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. Thus, AAV gene therapies are likely to alter the treatment paradigm for hemophilia A and B. © 2019 by The American Society of Hematology. https://doi.org/10.1016/j.biotechadv.2019.107502. Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patient’s own defective gene. AT number 154 4214 33. These oncology-directed therapies will compete with more traditional modalities (many of which will soon have biosimilar competition ), and thus will need to demonstrate increased cost-effectiveness. doi: https://doi.org/10.1182/hematology.2019000007. This study has been followed by unparalleled advances in gene therapy for hemophilia A and B, leading to clotting factor activity approaching normal or near-normal levels associated with a “zero bleed rates” in previously severely affected patients following a single administration of AAV vectors. Gene Therapy in 2019: Milestones and Challenges F or as long as medical researchers have been able to identify the genes responsible for particular diseases, they have set their sights on using that knowledge to devise gene therapies—ways to repair or circumvent the abnormal gene expression—to prevent or … 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. Ultimately, the first gene therapy trial was a success and the young patient was able to live a normal life outside of isolation. The hemophilias are ideally suited for gene therapy because a small increment in blood factor levels (≥5% of normal) is associated with significant amelioration of bleeding phenotype in severely affected patients. This review explores recent progress and the remaining limitations that need to be overcome for wider availability of this novel treatment of inherited bleeding disorders. Investigators are advancing with cautious optimism that effective, durable, and safe therapies will provide benefit to patients—not only those with single-gene disorders but those with complex acquired diseases as well. But the field suffered a major setback eight years later when an 18-year-old patient in a gene therapy study died after suffering a severe immune reaction to his treatment. 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. ScienceDirect ® is a registered trademark of Elsevier B.V. ScienceDirect ® is a registered trademark of Elsevier B.V. Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. All rights reserved. Review Article Gene Replacement Therapy: A Primer for the Health-system Pharmacist John Petrich, MS1, Dominic Marchese, PharmD2, Chris Jenkins, RBP3, Michael Storey, PharmD4, and Jill Blind, PharmD4 Abstract Purpose: Comprehensive review of gene replacement therapy with guidance and expert opinion on handling and administration for pharmacists. 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. Copyright ©2020 by American Society of Hematology, https://doi.org/10.1182/hematology.2019000007. By continuing you agree to the use of cookies. 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. The timeline of approved gene therapy drugs was shown in Fig. These authors contributed equally to this work. Vigene Biosciences, Inc . ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Search for other works by this author on: Mortality caused by intracranial bleeding in non-severe hemophilia A patients: reply, Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia, Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV, Practical aspects of extended half-life products for the treatment of haemophilia, BIVV001: The first investigational factor VIII therapy to break through the VWF ceiling in hemophilia A, with potential for extended protection for one week or longer, Factor VIII-mimetic function of humanized bispecific antibody in hemophilia A, Emicizumab prophylaxis in hemophilia A with inhibitors, An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia, A randomized trial of safety, pharmacokinetics and pharmacodynamics of concizumab in people with hemophilia A, Prophylactic infusion regimens in the management of hemophilia, Factor VIII Transkaryotic Therapy Study Group, Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A, Implantation of non-viral ex vivo genetically modified autologous dermal fibroblasts that express B-domain deleted human factor VIII in 12 severe hemophilia A study subjects, Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients, Ham-Wasserman Lecture: Hemophilia and related bleeding disorders: a story of dismay and success, AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B, Immune responses to AAV and to factor IX in a phase I study of AAV-mediated liver-directed gene transfer for hemophilia B, Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA, Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver, Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates, Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods, Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors, Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy, Adeno-associated mediated gene transfer for hemophilia B: 8 year follow up and impact of removing “empty viral particles” on safety and efficacy of gene transfer, Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Gene therapy for hemophilia: what does the future hold? Department of Haematology, UCL Cancer Institute, Katharine Dormandy Haemophilia and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, United Kingdom; and Freeline Therapeutics Ltd., Stevenage, United Kingdom. In 2011, the St. Jude/UCL phase 1/2 trial was the first to provide clear evidence of a stable dose-dependent increase in FIX levels in patients with severe hemophilia B following a single administration of adeno-associated viral (AAV) vectors. The purpose of the Cell and Gene Therapy Catapult UK Clinical Trials Database , Great Maze Pond, ondon, S1 9T sciencedirect ® is a registered trademark of Elsevier B.V. or licensors... Do not currently have access to this content the use of cookies the approved gene therapy drugs worldwide from... 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