{"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\/hi\/pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study\/","title":{"rendered":"PepGen \u0928\u0947 \u0918\u094b\u0937\u0923\u093e \u0915\u0940 \u0915\u093f \u0935\u0939 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0915\u094b \u0930\u094b\u0915 \u0926\u0947\u0917\u093e"},"content":{"rendered":"<p class=\"wp-block-paragraph\">PepGen, \u090f\u0915 \u0915\u094d\u0932\u0940\u0928\u093f\u0915\u0932-\u0938\u094d\u091f\u0947\u091c \u092c\u093e\u092f\u094b\u091f\u0947\u0915\u094d\u0928\u094b\u0932\u0949\u091c\u0940 \u0915\u0902\u092a\u0928\u0940 \u091c\u094b \u0911\u0932\u093f\u0917\u094b\u0928\u094d\u092f\u0941\u0915\u094d\u0932\u093f\u092f\u094b\u091f\u093e\u0907\u0921 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e \u0930\u0939\u0940 \u0939\u0948, \u0928\u0947 \u0906\u091c \u0918\u094b\u0937\u0923\u093e \u0915\u0940 \u0915\u093f \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0932\u093f\u090f \u0909\u0924\u094d\u0924\u0930\u0926\u093e\u092f\u0940 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (DMD) \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 PGN-EDO51 \u0915\u0940 \u091c\u093e\u0902\u091a \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0947 \u0905\u092a\u0928\u0947 CONNECT1-EDO51 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 10 mg\/kg \u0938\u092e\u0942\u0939 \u092e\u0947\u0902 \u092e\u093e\u092a\u0947 \u0917\u090f \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0947 \u0938\u094d\u0924\u0930\u094b\u0902 \u0915\u0947 \u0906\u0927\u093e\u0930 \u092a\u0930, \u0915\u0902\u092a\u0928\u0940 \u0905\u092a\u0928\u0947 \u0906\u0936\u093e\u091c\u0928\u0915 \u092e\u093e\u092f\u094b\u091f\u094b\u0928\u093f\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u091f\u093e\u0907\u092a 1 (DM1) \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0930\u092e \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u092a\u0930 \u0927\u094d\u092f\u093e\u0928 \u0915\u0947\u0902\u0926\u094d\u0930\u093f\u0924 \u0915\u0930\u0947\u0917\u0940 \u091c\u094b \u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u092e\u0947\u0902 \u091a\u0930\u0923 2 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0935\u093f\u0915\u093e\u0938 \u092e\u0947\u0902 \u0939\u0948\u0964 \u0915\u0902\u092a\u0928\u0940 \u0938\u094d\u0935\u0947\u091a\u094d\u091b\u093e \u0938\u0947 PGN-EDO51 \u0915\u0947 \u0935\u093f\u0915\u093e\u0938 \u0915\u094b \u092c\u0902\u0926 \u0915\u0930 \u0930\u0939\u0940 \u0939\u0948 \u0914\u0930 DMD \u0938\u0947 \u0938\u0902\u092c\u0902\u0927\u093f\u0924 \u0938\u092d\u0940 \u0936\u094b\u0927 \u0914\u0930 \u0935\u093f\u0915\u093e\u0938 \u0917\u0924\u093f\u0935\u093f\u0927\u093f\u092f\u094b\u0902 \u0915\u094b \u092c\u0902\u0926 \u0915\u0930\u0928\u0947 \u0915\u093e \u0907\u0930\u093e\u0926\u093e \u0930\u0916\u0924\u0940 \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CONNECT1 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 10 \u092e\u093f\u0932\u0940\u0917\u094d\u0930\u093e\u092e\/\u0915\u093f\u0917\u094d\u0930\u093e \u0938\u092e\u0942\u0939 (n=4) \u092e\u0947\u0902, PGN-EDO51 \u0928\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u094d\u0921 \u091f\u094d\u0930\u093e\u0902\u0938\u0915\u094d\u0930\u093f\u092a\u094d\u091f \u0915\u094b 4.26% (3.5% \u0915\u0940 \u0914\u0938\u0924 \u0935\u0943\u0926\u094d\u0927\u093f) \u0924\u0915 \u092c\u0922\u093c\u093e \u0926\u093f\u092f\u093e; \u0939\u093e\u0932\u093e\u0901\u0915\u093f, \u0915\u0941\u0932 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u0915\u0947\u0935\u0932 \u0938\u093e\u092e\u093e\u0928\u094d\u092f \u0938\u094d\u0924\u0930 \u0915\u0947 0.59% (0.36% \u0915\u0940 \u0914\u0938\u0924 \u0935\u0943\u0926\u094d\u0927\u093f) \u0924\u0915 \u092c\u0922\u093c\u093e\u0964 PGN-EDO51 \u0915\u0940 \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093c\u093e\u0907\u0932 \u0906\u092e \u0924\u094c\u0930 \u092a\u0930 \u0905\u0928\u0941\u0915\u0942\u0932 \u092c\u0928\u0940 \u0930\u0939\u0940 \u0914\u0930 \u0909\u092a\u091a\u093e\u0930 \u0938\u0947 \u0938\u0902\u092c\u0902\u0927\u093f\u0924 \u0938\u092d\u0940 \u092a\u094d\u0930\u0924\u093f\u0915\u0942\u0932 \u0918\u091f\u0928\u093e\u090f\u0901 \u092a\u094d\u0930\u0915\u0943\u0924\u093f \u092e\u0947\u0902 \u0939\u0932\u094d\u0915\u0940 \u0925\u0940\u0902\u0964 \u0905\u0927\u094d\u092f\u092f\u0928 \u092e\u0947\u0902 \u0915\u094b\u0908 \u0917\u0902\u092d\u0940\u0930 \u092a\u094d\u0930\u0924\u093f\u0915\u0942\u0932 \u0918\u091f\u0928\u093e \u0930\u093f\u092a\u094b\u0930\u094d\u091f \u0928\u0939\u0940\u0902 \u0915\u0940 \u0917\u0908\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0914\u0930 \u092a\u0922\u093c\u0947\u0902:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/hi\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">\u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0940 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u0940 \u0917\u0908<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CONNECT1 \u0915\u0947 \u0905\u0927\u094d\u092f\u0915\u094d\u0937 \u0914\u0930 \u0938\u0940\u0908\u0913, \u092a\u0940\u090f\u091a\u0921\u0940, \u091c\u0947\u092e\u094d\u0938 \u092e\u0948\u0915\u0906\u0930\u094d\u0925\u0930 \u0928\u0947 \u0915\u0939\u093e, &quot;10 \u092e\u093f\u0932\u0940\u0917\u094d\u0930\u093e\u092e\/\u0915\u093f\u0932\u094b\u0917\u094d\u0930\u093e\u092e \u0916\u0941\u0930\u093e\u0915 \u0935\u093e\u0932\u0947 \u0938\u092e\u0942\u0939 \u092e\u0947\u0902 CONNECT1 \u092e\u0947\u0902 \u0926\u0947\u0916\u0947 \u0917\u090f \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0938\u0947 \u0939\u092e \u0928\u093f\u0930\u093e\u0936 \u0939\u0948\u0902, \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u0939\u092e\u0947\u0902 \u0909\u092e\u094d\u092e\u0940\u0926 \u0925\u0940 \u0915\u093f \u0939\u092e \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u092e\u094c\u091c\u0942\u0926\u093e \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u092e\u0947\u0902 \u0914\u0930 \u0905\u0927\u093f\u0915 \u0917\u0939\u0928 \u0924\u0930\u0940\u0915\u0947 \u0938\u0947 \u0938\u0941\u0927\u093e\u0930 \u0915\u0930 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\u0964&quot; &quot;\u091c\u0948\u0938\u0947 \u0939\u0940 \u0939\u092e \u0905\u092a\u0928\u0947 DMD \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0930\u092e \u0915\u094b \u0938\u092e\u093e\u092a\u094d\u0924 \u0915\u0930 \u0930\u0939\u0947 \u0939\u0948\u0902, \u0939\u092e \u0907\u0938 \u0936\u094b\u0927 \u092e\u0947\u0902 \u0909\u0928\u0915\u0947 \u0938\u092e\u0930\u094d\u0925\u0928 \u0914\u0930 \u092d\u093e\u0917\u0940\u0926\u093e\u0930\u0940 \u0915\u0947 \u0932\u093f\u090f \u0930\u094b\u0917\u093f\u092f\u094b\u0902, \u092a\u0930\u093f\u0935\u093e\u0930\u094b\u0902, \u0926\u0947\u0916\u092d\u093e\u0932 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u094b\u0902, \u091c\u093e\u0902\u091a\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0914\u0930 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0930\u094d\u092e\u091a\u093e\u0930\u093f\u092f\u094b\u0902 \u0915\u094b \u0927\u0928\u094d\u092f\u0935\u093e\u0926 \u0926\u0947\u0928\u093e \u091a\u093e\u0939\u0924\u0947 \u0939\u0948\u0902\u0964 \u092e\u0948\u0902 DMD \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0928\u090f \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0939\u092e\u093e\u0930\u0940 \u091f\u0940\u092e \u0915\u0940 \u0915\u0921\u093c\u0940 \u092e\u0947\u0939\u0928\u0924 \u0914\u0930 \u092a\u094d\u0930\u0924\u093f\u092c\u0926\u094d\u0927\u0924\u093e \u0915\u094b \u092d\u0940 \u0938\u094d\u0935\u0940\u0915\u093e\u0930 \u0915\u0930\u0928\u093e \u091a\u093e\u0939\u0924\u093e \u0939\u0942\u0902\u0964&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0914\u0930 \u092a\u0922\u093c\u0947\u0902<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/hi\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0915\u0947 \u0932\u093f\u090f \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 (\u0938\u092d\u0940 \u0936\u094b\u0927\u094b\u0902 \u0915\u0940 \u0938\u0942\u091a\u0940)<\/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\/hi\/wp-json\/wp\/v2\/posts\/3883","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/comments?post=3883"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3883\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/3889"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=3883"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=3883"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=3883"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}