{"id":2961,"date":"2025-02-18T15:46:12","date_gmt":"2025-02-18T12:46:12","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2961"},"modified":"2025-03-07T12:57:05","modified_gmt":"2025-03-07T09:57:05","slug":"solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003\/","title":{"rendered":"Solid Biosciences publie des donn\u00e9es cliniques initiales positives sur le candidat \u00e0 la th\u00e9rapie g\u00e9nique de nouvelle g\u00e9n\u00e9ration pour la maladie de Duchenne, SGT-003"},"content":{"rendered":"

Solid Biosciences, une soci\u00e9t\u00e9 sp\u00e9cialis\u00e9e dans les sciences de la vie qui d\u00e9veloppe des m\u00e9dicaments g\u00e9n\u00e9tiques de pr\u00e9cision pour les maladies neuromusculaires et cardiaques, a annonc\u00e9 aujourd'hui des donn\u00e9es initiales positives issues de l'essai de phase 1\/2 INSPIRE DUCHENNE \u00e9valuant SGT-003, un produit candidat de th\u00e9rapie g\u00e9nique de nouvelle g\u00e9n\u00e9ration destin\u00e9 au traitement de la dystrophie musculaire de Duchenne (Duchenne). Les donn\u00e9es interm\u00e9diaires de biopsie \u00e0 90 jours rapport\u00e9es chez les trois premiers participants ont montr\u00e9 une expression moyenne de microdystrophine de 110%, telle que mesur\u00e9e par Western blot, et des am\u00e9liorations de plusieurs biomarqueurs qui sont des indicateurs de la sant\u00e9 et de la r\u00e9silience musculaires.<\/p>\n\n\n\n

Donn\u00e9es cliniques du candidat \u00e0 la th\u00e9rapie g\u00e9nique de la maladie de Duchenne SGT-003<\/h2>\n\n\n\n
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  1. L'expression moyenne de microdystrophine de 110% (N = 3) et les am\u00e9liorations significatives observ\u00e9es dans plusieurs biomarqueurs suppl\u00e9mentaires de la sant\u00e9 musculaire soutiennent le potentiel de SGT-003 en tant que candidat \u00e0 la th\u00e9rapie g\u00e9nique de la dystrophie musculaire de Duchenne de nouvelle g\u00e9n\u00e9ration et de premier ordre.<\/li>\n\n\n\n
  2. Des signes pr\u00e9coces encourageants d\u2019un b\u00e9n\u00e9fice cardiaque potentiel ont \u00e9t\u00e9 observ\u00e9s.<\/li>\n\n\n\n
  3. Le SGT-003 a \u00e9t\u00e9 bien tol\u00e9r\u00e9 chez les 6 participants trait\u00e9s au 11 f\u00e9vrier 2025, sans aucun \u00e9v\u00e9nement ind\u00e9sirable grave observ\u00e9.<\/li>\n\n\n\n
  4. Le recrutement des participants se poursuit, le 7e participant ayant \u00e9t\u00e9 dos\u00e9 le 17 f\u00e9vrier 2025 ; la soci\u00e9t\u00e9 pr\u00e9voit de doser environ 20 participants au total d'ici le quatri\u00e8me trimestre 2025.<\/li>\n<\/ol>","protected":false},"excerpt":{"rendered":"

    Solid Biosciences, a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced positive initial data from the Phase 1\/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data reported in the first three participants showed […]<\/p>\n","protected":false},"author":1,"featured_media":2963,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[137,110,170,109],"class_list":["post-2961","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trial","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"subtitle":"Solid Biosciences, a clinical-stage biotechnology company, shared positive initial data from its Phase 1\/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2961","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=2961"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2961\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/2963"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=2961"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=2961"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=2961"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}