LAMA2-CMD est une maladie caract\u00e9ris\u00e9e par l’absence de la prot\u00e9ine LAMA2, entra\u00eenant une dystrophie musculaire s\u00e9v\u00e8re d\u00e8s le d\u00e9but de la vie. La FDA am\u00e9ricaine a accord\u00e9 le statut de m\u00e9dicament orphelin \u00e0 la th\u00e9rapie exp\u00e9rimentale MDL-101 de Modalis Therapeutics, destin\u00e9e \u00e0 traiter cette maladie.<\/p>\n
MDL-101 est une th\u00e9rapie d’\u00e9dition \u00e9pig\u00e9n\u00e9tique qui cible le g\u00e8ne LAMA1, un proche homologue du g\u00e8ne LAMA2 responsable de la maladie. Elle utilise une version modifi\u00e9e de l’enzyme Cas9, combin\u00e9e \u00e0 un domaine activateur, et est administr\u00e9e via un vecteur viral sp\u00e9cifique aux muscles.<\/p>\n
Cette th\u00e9rapie vise \u00e0 augmenter les produits du g\u00e8ne LAMA1 dans les tissus musculaires des patients pour compenser le dysfonctionnement caus\u00e9 par les mutations de LAMA2. Elle pourrait offrir un traitement unique et durable pour les personnes atteintes de LAMA2-CMD.<\/p>\n
Le statut de m\u00e9dicament orphelin aide au d\u00e9veloppement de traitements pour des maladies touchant moins de 200 000 patients aux \u00c9tats-Unis. Pour MDL-101, cela inclut des exemptions de frais d’application, des r\u00e9ductions fiscales pour le d\u00e9veloppement clinique et des avantages pour la promotion du produit.<\/p>\n
Si approuv\u00e9, MDL-101 b\u00e9n\u00e9ficierait de sept ans d’exclusivit\u00e9 commerciale sur le march\u00e9 am\u00e9ricain. La FDA a \u00e9galement attribu\u00e9 une d\u00e9signation pour maladie p\u00e9diatrique rare \u00e0 cette th\u00e9rapie g\u00e9nique pour LAMA2-CMD.<\/p>\n
Modalis d\u00e9veloppe des th\u00e9rapies g\u00e9n\u00e9tiques de pr\u00e9cision gr\u00e2ce \u00e0 sa technologie d’\u00e9dition \u00e9pig\u00e9nomique, CRISPR-GNDM, qui permet de modifier l’expression des g\u00e8nes sans casser l’ADN.<\/p>\n
Enfin, l’industrie des th\u00e9rapies cellulaires et g\u00e9niques conna\u00eet une croissance rapide, promettant de transformer le traitement des maladies.<\/p>\n","protected":false},"excerpt":{"rendered":"
LAMA2-CMD est une maladie caract\u00e9ris\u00e9e par l’absence de la prot\u00e9ine LAMA2, entra\u00eenant une dystrophie musculaire s\u00e9v\u00e8re d\u00e8s le d\u00e9but de la vie. La FDA am\u00e9ricaine a accord\u00e9 le statut de m\u00e9dicament orphelin \u00e0 la th\u00e9rapie exp\u00e9rimentale MDL-101 de Modalis Therapeutics, destin\u00e9e \u00e0 traiter cette maladie. MDL-101 est une th\u00e9rapie d’\u00e9dition \u00e9pig\u00e9n\u00e9tique qui cible le g\u00e8ne […]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[684],"tags":[],"class_list":["post-69570","post","type-post","status-publish","format-standard","hentry","category-nouvelles-pharmaceutiques"],"_links":{"self":[{"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/posts\/69570","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/comments?post=69570"}],"version-history":[{"count":1,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/posts\/69570\/revisions"}],"predecessor-version":[{"id":69573,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/posts\/69570\/revisions\/69573"}],"wp:attachment":[{"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/media?parent=69570"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/categories?post=69570"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.pharmacie-vezere.com\/promotions\/wp-json\/wp\/v2\/tags?post=69570"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}