{"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":"2026-03-12T10:11:29","modified_gmt":"2026-03-12T14:11:29","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 – are we ready now?"},"content":{"rendered":"\t\t
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At the WFH 2022 World Congress in Montreal, Canada, the plenary \u201cGene therapy – 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>

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>

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>

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>

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\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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WFH 2022 World Congress<\/h3>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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\n\t\t\t\t\tMay 8 to 11, 2022<\/span>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Register now!<\/strong><\/a><\/p>\n\t\t\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":"

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":[169,33],"tags":[],"search-category":[85],"class_list":["post-37399","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-events-2","category-wfh-world-congress","search-category-article"],"yoast_head":"\nPlenary: Gene therapy - are we ready now? | FMH - F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Plenary: Gene therapy - are we ready now?\" \/>\n<meta property=\"og:description\" content=\"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.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\" \/>\n<meta property=\"og:site_name\" content=\"FMH - F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/wfhemophilia\/\" \/>\n<meta property=\"article:published_time\" content=\"2022-05-10T18:18:05+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-03-12T14:11:29+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/wfh.org\/wp-content\/uploads\/2022\/05\/news-plenary-gene-therapy-may10.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1200\" \/>\n\t<meta property=\"og:image:height\" content=\"700\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Electra Rozakis\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"\u00c9crit par\" \/>\n\t<meta name=\"twitter:data1\" content=\"Electra Rozakis\" \/>\n\t<meta name=\"twitter:label2\" content=\"Dur\u00e9e de lecture estim\u00e9e\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\"},\"author\":{\"name\":\"Electra Rozakis\",\"@id\":\"https:\/\/wfh.org\/fr\/#\/schema\/person\/622f4b0ed56b8e4a987dd20bef774887\"},\"headline\":\"Plenary: Gene therapy – are we ready now?\",\"datePublished\":\"2022-05-10T18:18:05+00:00\",\"dateModified\":\"2026-03-12T14:11:29+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\"},\"wordCount\":339,\"publisher\":{\"@id\":\"https:\/\/wfh.org\/fr\/#organization\"},\"image\":{\"@id\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/wfh.org\/wp-content\/uploads\/2022\/05\/news-plenary-gene-therapy-may10.jpg\",\"articleSection\":[\"Events\",\"WFH World Congress\"],\"inLanguage\":\"fr-FR\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\",\"url\":\"https:\/\/wfh.org\/fr\/article\/plenary-gene-therapy-are-we-ready-now\/\",\"name\":\"Plenary: Gene therapy - are we ready now? 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