{"id":3883,"date":"2025-05-29T11:06:14","date_gmt":"2025-05-29T08:06:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3883"},"modified":"2025-11-03T20:23:45","modified_gmt":"2025-11-03T17:23:45","slug":"pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study\/","title":{"rendered":"\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 PepGen \u0623\u0646\u0647\u0627 \u0633\u062a\u0648\u0642\u0641 \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u062f\u0631\u0627\u0633\u0629 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0623\u0639\u0644\u0646\u062a \u0627\u0644\u064a\u0648\u0645 \u0634\u0631\u0643\u0629 PepGen\u060c \u0648\u0647\u064a \u0634\u0631\u0643\u0629 \u062a\u0643\u0646\u0648\u0644\u0648\u062c\u064a\u0627 \u062d\u064a\u0648\u064a\u0629 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u062a\u0639\u0645\u0644 \u0639\u0644\u0649 \u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u0642\u0627\u062f\u0645 \u0645\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u0623\u0648\u0644\u064a\u063a\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a\u060c \u0623\u0646\u0647 \u0628\u0646\u0627\u0621\u064b \u0639\u0644\u0649 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0645\u064f\u0642\u0627\u0633\u0629 \u0641\u064a \u0645\u062c\u0645\u0648\u0639\u0629 10 \u0645\u0644\u063a\/\u0643\u063a \u0645\u0646 \u062f\u0631\u0627\u0633\u0629 CONNECT1-EDO51 \u0627\u0644\u062a\u064a \u0623\u062c\u0631\u062a\u0647\u0627 \u0627\u0644\u0634\u0631\u0643\u0629 \u0639\u0644\u0649 PGN-EDO51 \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD) \u0627\u0644\u0630\u064a\u0646 \u064a\u064f\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u0633\u062a\u064f\u0631\u0643\u0632 \u0627\u0644\u0634\u0631\u0643\u0629 \u0639\u0644\u0649 \u062a\u0637\u0648\u064a\u0631 \u0628\u0631\u0646\u0627\u0645\u062c\u0647\u0627 \u0627\u0644\u0648\u0627\u0639\u062f \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0645\u0646 \u0627\u0644\u0646\u0648\u0639 \u0627\u0644\u0623\u0648\u0644 (DM1)\u060c \u0648\u0627\u0644\u0630\u064a \u064a\u0645\u0631 \u062d\u0627\u0644\u064a\u064b\u0627 \u0628\u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u062b\u0627\u0646\u064a\u0629 \u0645\u0646 \u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a. \u0648\u062a\u062a\u0648\u0642\u0641 \u0627\u0644\u0634\u0631\u0643\u0629 \u0637\u0648\u0627\u0639\u064a\u0629\u064b \u0639\u0646 \u062a\u0637\u0648\u064a\u0631 PGN-EDO51\u060c \u0648\u062a\u0639\u062a\u0632\u0645 \u0625\u0646\u0647\u0627\u0621 \u062c\u0645\u064a\u0639 \u0623\u0646\u0634\u0637\u0629 \u0627\u0644\u0628\u062d\u062b \u0648\u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0645\u062a\u0639\u0644\u0642\u0629 \u0628\u0640 DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0641\u064a \u0645\u062c\u0645\u0648\u0639\u0629 10 \u0645\u0644\u063a\/\u0643\u063a (\u0646=4) \u0645\u0646 \u062f\u0631\u0627\u0633\u0629 CONNECT1\u060c \u0632\u0627\u062f PGN-EDO51 \u0645\u0646 \u0646\u064f\u0633\u062e \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0627\u0644\u0645\u064f\u062a\u062e\u0637\u0627\u0629 \u0625\u0644\u0649 4.26% (\u0628\u0645\u062a\u0648\u0633\u0637 \u0632\u064a\u0627\u062f\u0629 3.5%)\u061b \u0648\u0645\u0639 \u0630\u0644\u0643\u060c \u0644\u0645 \u064a\u0631\u062a\u0641\u0639 \u0625\u062c\u0645\u0627\u0644\u064a \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0625\u0644\u0627 \u0625\u0644\u0649 0.59% \u0645\u0646 \u0627\u0644\u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0627\u0644\u0637\u0628\u064a\u0639\u064a\u0629 (\u0628\u0645\u062a\u0648\u0633\u0637 \u0632\u064a\u0627\u062f\u0629 0.36%). \u0638\u0644 \u0645\u0644\u0641 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0644\u0640 PGN-EDO51 \u0625\u064a\u062c\u0627\u0628\u064a\u064b\u0627 \u0628\u0634\u0643\u0644 \u0639\u0627\u0645\u060c \u0648\u0643\u0627\u0646\u062a \u062c\u0645\u064a\u0639 \u0627\u0644\u0622\u062b\u0627\u0631 \u0627\u0644\u062c\u0627\u0646\u0628\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u062e\u0641\u064a\u0641\u0629 \u0628\u0637\u0628\u064a\u0639\u062a\u0647\u0627. \u0644\u0645 \u062a\u064f\u0633\u062c\u0644 \u0623\u064a \u0622\u062b\u0627\u0631 \u062c\u0627\u0646\u0628\u064a\u0629 \u062e\u0637\u064a\u0631\u0629 \u0641\u064a \u0627\u0644\u062f\u0631\u0627\u0633\u0629.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/ar\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">\u062a\u0645 \u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u0642\u0627\u062f\u0645 \u0645\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0642\u0627\u0644 \u0627\u0644\u062f\u0643\u062a\u0648\u0631 \u062c\u064a\u0645\u0633 \u0645\u0627\u0643 \u0622\u0631\u062b\u0631\u060c \u0627\u0644\u0631\u0626\u064a\u0633 \u0648\u0627\u0644\u0645\u062f\u064a\u0631 \u0627\u0644\u062a\u0646\u0641\u064a\u0630\u064a \u0644\u0634\u0631\u0643\u0629 PepGen: &quot;\u0646\u0634\u0639\u0631 \u0628\u062e\u064a\u0628\u0629 \u0623\u0645\u0644 \u0625\u0632\u0627\u0621 \u0646\u062a\u0627\u0626\u062c \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u062a\u064a \u0644\u0648\u062d\u0638\u062a \u0641\u064a \u0645\u062c\u0645\u0648\u0639\u0629 \u062c\u0631\u0639\u0627\u062a 10 \u0645\u0644\u063a\/\u0643\u063a \u0641\u064a \u062a\u062c\u0631\u0628\u0629 CONNECT1\u060c \u0625\u0630 \u0643\u0646\u0627 \u0646\u0623\u0645\u0644 \u0623\u0646 \u0646\u062a\u0645\u0643\u0646 \u0645\u0646 \u062a\u062d\u0633\u064a\u0646 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062d\u0627\u0644\u064a\u0629 \u0644\u0644\u0645\u0631\u0636\u0649 \u0628\u0634\u0643\u0644 \u0623\u0639\u0645\u0642&quot;. \u0648\u0623\u0636\u0627\u0641: &quot;\u0645\u0639 \u0627\u0642\u062a\u0631\u0627\u0628\u0646\u0627 \u0645\u0646 \u0646\u0647\u0627\u064a\u0629 \u0628\u0631\u0646\u0627\u0645\u062c\u0646\u0627 \u0644\u0639\u0644\u0627\u062c \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u0646\u0648\u062f \u0623\u0646 \u0646\u0634\u0643\u0631 \u0627\u0644\u0645\u0631\u0636\u0649 \u0648\u0639\u0627\u0626\u0644\u0627\u062a\u0647\u0645 \u0648\u0645\u0642\u062f\u0645\u064a \u0627\u0644\u0631\u0639\u0627\u064a\u0629 \u0648\u0627\u0644\u0628\u0627\u062d\u062b\u064a\u0646 \u0648\u0641\u0631\u064a\u0642 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0639\u0644\u0649 \u062f\u0639\u0645\u0647\u0645 \u0648\u0645\u0634\u0627\u0631\u0643\u062a\u0647\u0645 \u0641\u064a \u0647\u0630\u0627 \u0627\u0644\u0628\u062d\u062b. \u0643\u0645\u0627 \u0623\u0648\u062f \u0623\u0646 \u0623\u0634\u064a\u062f \u0628\u0627\u0644\u0639\u0645\u0644 \u0627\u0644\u062c\u0627\u062f \u0644\u0641\u0631\u064a\u0642\u0646\u0627 \u0648\u0627\u0644\u062a\u0632\u0627\u0645\u0647 \u0628\u062a\u0637\u0648\u064a\u0631 \u0639\u0644\u0627\u062c\u0627\u062a \u062c\u062f\u064a\u062f\u0629 \u0645\u062d\u062a\u0645\u0644\u0629 \u0644\u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a&quot;.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/ar\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 (\u0642\u0627\u0626\u0645\u0629 \u0628\u062c\u0645\u064a\u0639 \u0627\u0644\u0623\u0628\u062d\u0627\u062b)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg\/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3889,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,275,122,193,194,371,273,274],"class_list":["post-3883","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-connect1-edo51","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-freedom-dm1","tag-pepgen","tag-pgn-edo51"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3883","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/comments?post=3883"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3883\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3889"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3883"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3883"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3883"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}