{"id":37399,"date":"2022-05-10T14:18:05","date_gmt":"2022-05-10T18:18:05","guid":{"rendered":"https:\/\/wfh.org\/plenary-gene-therapy-are-we-ready-now\/"},"modified":"2024-04-11T08:35:20","modified_gmt":"2024-04-11T12:35:20","slug":"plenary-gene-therapy-are-we-ready-now","status":"publish","type":"post","link":"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/","title":{"rendered":"Plenary: Gene therapy &#8211; are we ready now?"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-post\" data-elementor-id=\"37399\" class=\"elementor elementor-37399 elementor-37288\" data-elementor-post-type=\"post\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-57238ed elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"57238ed\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-5d4f5ed\" data-id=\"5d4f5ed\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-bc649f6 elementor-widget elementor-widget-text-editor\" data-id=\"bc649f6\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-widget-text-editor.elementor-drop-cap-view-stacked .elementor-drop-cap{background-color:#69727d;color:#fff}.elementor-widget-text-editor.elementor-drop-cap-view-framed .elementor-drop-cap{color:#69727d;border:3px solid;background-color:transparent}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap{margin-top:8px}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap-letter{width:1em;height:1em}.elementor-widget-text-editor .elementor-drop-cap{float:left;text-align:center;line-height:1;font-size:50px}.elementor-widget-text-editor .elementor-drop-cap-letter{display:inline-block}<\/style>\t\t\t\t<p class=\"\">At the WFH 2022 World Congress in Montreal, Canada, the plenary \u201cGene therapy &#8211; are we ready now?\u201d looked at what our prospects are with this exciting new medical avenue. The event\u2019s speaker was Radek Kaczmarek, Postdoctoral Research Associate, Gene and Cell Therapy Group, Wells Center for Pediatric Research, Indiana University School of Medicine, U.S.A, and WFH Coagulation Product Safety, Supply, and Access (CPSSA) Committee chair (Poland\/U.S.A.).<\/p>\r\n<p class=\"\">Kaczmarek covered a brief history of hemophilia treatment, from whole blood and plasma treatment, to cryoprecipitate, non-factor products, and gene therapy. He said the questions we must ask ourselves about gene therapy revolve around variability, reliability, durability, tolerability, and visibility (transparency). Kaczmarek also covered core outcomes, such as frequency of bleeds, factor activity level, duration of expression, chronic pain, utilization of the healthcare system, and the mental health of the patient.<\/p>\r\n<p class=\"\">The advance of gene therapies toward commercialization has outpaced our fundamental biological understanding of AAV gene transfer in humans. The issues that now need more attention are pre-existing immunity, immune responses, and immunosuppression; degradation of AAV capsid load; unfolded protein response (FVIII); poor transduction efficiency (necessitates high vector doses); data sharing; vector integration; standardization of assay; and reducing uncertainty. <\/p>\r\n<p class=\"\">Collaboration is critical to a successful outcome for gene therapy, says Kaczmarek, which is why it\u2019s significant that a core outcome data set to facilitate the lifelong monitoring of safety, efficacy, and durability outcomes has been agreed upon and used to develop the WFH Gene Therapy Registry by the WFH in collaboration with ISTH SSC, EHC, NHF, ATHN, industry partners and regulatory partners.<\/p>\r\n<p class=\"\">A big question: does the state of the art match the dream? Kaczmarek says it might, but several patient subgroups miss out, such as females, children, inhibitors, people with pre-existing immunity, those who are geographically disadvantaged, etc. All of this presents a unique challenge for healthcare practitioners (HCPs) who will be introducing and delivering it to the patients.<\/p>\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-f8b66a3 elementor-widget elementor-widget-spacer\" data-id=\"f8b66a3\" data-element_type=\"widget\" data-widget_type=\"spacer.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-column .elementor-spacer-inner{height:var(--spacer-size)}.e-con{--container-widget-width:100%}.e-con-inner>.elementor-widget-spacer,.e-con>.elementor-widget-spacer{width:var(--container-widget-width,var(--spacer-size));--align-self:var(--container-widget-align-self,initial);--flex-shrink:0}.e-con-inner>.elementor-widget-spacer>.elementor-widget-container,.e-con>.elementor-widget-spacer>.elementor-widget-container{height:100%;width:100%}.e-con-inner>.elementor-widget-spacer>.elementor-widget-container>.elementor-spacer,.e-con>.elementor-widget-spacer>.elementor-widget-container>.elementor-spacer{height:100%}.e-con-inner>.elementor-widget-spacer>.elementor-widget-container>.elementor-spacer>.elementor-spacer-inner,.e-con>.elementor-widget-spacer>.elementor-widget-container>.elementor-spacer>.elementor-spacer-inner{height:var(--container-widget-height,var(--spacer-size))}.e-con-inner>.elementor-widget-spacer.elementor-widget-empty,.e-con>.elementor-widget-spacer.elementor-widget-empty{position:relative;min-height:22px;min-width:22px}.e-con-inner>.elementor-widget-spacer.elementor-widget-empty .elementor-widget-empty-icon,.e-con>.elementor-widget-spacer.elementor-widget-empty .elementor-widget-empty-icon{position:absolute;top:0;bottom:0;left:0;right:0;margin:auto;padding:0;width:22px;height:22px}<\/style>\t\t<div class=\"elementor-spacer\">\n\t\t\t<div class=\"elementor-spacer-inner\"><\/div>\n\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-97738b2 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"97738b2\" data-element_type=\"section\" data-settings=\"{&quot;background_background&quot;:&quot;gradient&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-38090e2\" data-id=\"38090e2\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-ac55b80 elementor-widget elementor-widget-heading\" data-id=\"ac55b80\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-heading-title{padding:0;margin:0;line-height:1}.elementor-widget-heading .elementor-heading-title[class*=elementor-size-]>a{color:inherit;font-size:inherit;line-height:inherit}.elementor-widget-heading .elementor-heading-title.elementor-size-small{font-size:15px}.elementor-widget-heading .elementor-heading-title.elementor-size-medium{font-size:19px}.elementor-widget-heading .elementor-heading-title.elementor-size-large{font-size:29px}.elementor-widget-heading .elementor-heading-title.elementor-size-xl{font-size:39px}.elementor-widget-heading .elementor-heading-title.elementor-size-xxl{font-size:59px}<\/style><h3 class=\"elementor-heading-title elementor-size-default\">WFH 2022 World Congress<\/h3>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-ffdb32d elementor-widget elementor-widget-heading\" data-id=\"ffdb32d\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<span class=\"elementor-heading-title elementor-size-default\">May 8 to 11, 2022<\/span>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-f1a8eb4 elementor-widget elementor-widget-spacer\" data-id=\"f1a8eb4\" data-element_type=\"widget\" data-widget_type=\"spacer.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<div class=\"elementor-spacer\">\n\t\t\t<div class=\"elementor-spacer-inner\"><\/div>\n\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-65f9a0d elementor-widget elementor-widget-text-editor\" data-id=\"65f9a0d\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<p><a href=\"https:\/\/congress.wfh.org\/registration\/register-now\/\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>Register now!<\/strong><\/a><\/p>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>Hemophilia therapy has evolved from rudimentary transfusion-based approaches to an unprecedented level of innovation with the potential of a functional cure brought by gene therapy.<\/p>\n","protected":false},"author":3,"featured_media":37373,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[187],"tags":[],"search-category":[85],"class_list":["post-37399","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-congres-mondiale-de-la-fmh","search-category-article"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v23.4 (Yoast SEO v23.4) - 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