Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. Amit C. Nathwani; Gene therapy for hemophilia. 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. We strive to achieve our vision by combining new manufacturing technologies and high yield production cell lines with a rigorous quality control system for cGMP . Ultimately, the first gene therapy trial was a success and the young patient was able to live a normal life outside of isolation. 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 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. 2. All rights reserved. The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy, Unraveling the complex story of immune responses to AAV vectors trial after trial, Hemophilia B gene therapy with a high-specific-activity factor IX variant, Phase 2b trial of AMT-061 (AAV5-Padua hFIX): translation into humans of an enhanced gene transfer vector for adults with severe or moderate-severe hemophilia B, A single intravenous infusion of FLT180a results in factor IX activity levels of more than 40% and has the potential to provide a functional cure for patients with haemophilia B, Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant, AAV5-factor VIII gene transfer in severe hemophilia A, GO-8: preliminary results of a phase I/II dose escalation trial of gene therapy for haemophilia a using a novel human factor VIII variant, SPK-8011: Preliminary results from a phase 1/2 trial of investigational gene therapy for hemophilia A, Initial results of the Alta study, a phase 1/2, open label, adaptive, dose-ranging study to assess the safety and tolerability of SB-525 gene therapy in adult subjects with severe hemophilia A, Proceedings from ISTH 2019: Practice in Thrombosis and Hemostasis, X-linked thrombophilia with a mutant factor IX (factor IX Padua), Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver, Assessing the potential for AAV vector genotoxicity in a murine model, Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas, AAV vector integration sites in mouse hepatocellular carcinoma, Adeno-associated virus vectorology, manufacturing, and clinical applications, Toward exascale production of recombinant adeno-associated virus for gene transfer applications, Overcoming preexisting humoral immunity to AAV using capsid decoys, Genetic targeting of the albumin locus to treat hemophilia, Promoterless gene targeting without nucleases ameliorates haemophilia B in mice, In vivo genome editing of the albumin locus as a platform for protein replacement therapy, Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates. 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. 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. 07964711, with registered oce at 12th Floor Tower ing, Guys Hospital, Great Maze Pond, ondon, S1 9T. Gene Therapy Gathers Momentum Those who have followed the gene-therapy field over the decades may be weary of forward-looking positive statements. 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.

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