{"id":823783,"date":"2025-03-10T16:08:05","date_gmt":"2025-03-10T20:08:05","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/"},"modified":"2025-03-10T16:08:05","modified_gmt":"2025-03-10T20:08:05","slug":"pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/","title":{"rendered":"PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference"},"content":{"rendered":"<p>        <!--.bwalignc { text-align: center; list-style-position: inside }\n.bwuline { text-decoration: underline }body {font:normal small Arial,Helvetica,sans-serif;color:#000;background-color:#fff;padding:24px;margin:0;} a img {border:0;} h3 {font-size:medium;color:#000;margin:0 0 1em 0; text-align:center;}-->  <\/p>\n<p class=\"bwalignc\"><b>PepGen Announces Presentations at the 2025 Muscular Dystrophy Association <\/b><b>Clinical &amp; Scientific Conference<\/b><\/p>\n<p>BOSTON&#8211;(<a href=\"http:\/\/www.businesswire.com\">BUSINESS WIRE<\/a>)&#8211;<br \/>\nPepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently <a rel=\"nofollow\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Finvestors.pepgen.com%2Fnews-releases%2Fnews-release-details%2Fpepgen-announces-positive-initial-results-including-robust&amp;esheet=54221279&amp;newsitemid=20250310990282&amp;lan=en-US&amp;anchor=announced&amp;index=1&amp;md5=1b482526112c8480950f30a1c035956e\">announced<\/a>.\n<\/p>\n<p><b><span class=\"bwuline\">Oral Presentations<\/span><\/b><\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping<br \/>\n<br \/><b><span class=\"bwuline\">Session:<\/span><\/b> Clinical Trial Updates<br \/>\n<br \/><b><span class=\"bwuline\">Date &amp; Time:<\/span><\/b> Wednesday, March 19<sup>th<\/sup> at 8:45-9:00 a.m. CT<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Paul Streck, MD, MBA, Executive Vice President, R&amp;D\n<\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b>FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)<br \/>\n<br \/><b><span class=\"bwuline\">Session:<\/span><\/b> Clinical Trial Updates<br \/>\n<br \/><b><span class=\"bwuline\">Date &amp; Time:<\/span><\/b> Wednesday, March 19<sup>th<\/sup> at 12:45-1:00 p.m. CT<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Jane Larkindale, DPhil, Vice President, Clinical Science\n<\/p>\n<p><b><span class=\"bwuline\">Poster Presentations<\/span><\/b><\/p>\n<p><i>Posters will be displayed beginning at 6:00-8:00 p.m. CT on Sunday, March 16<sup>th<\/sup> through Tuesday, March 18<sup>th<\/sup> in the conference exhibit hall.<\/i><\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> Mechanistic Characterization of Enhanced Delivery Oligonucleotide (EDO) Platform<br \/>\n<br \/><b><span class=\"bwuline\">Poster Number:<\/span><\/b> #P21<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Jane Larkindale, DPhil, Vice President, Clinical Science\n<\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> Nonclinical Data for PGN-EDODM1 Demonstrated Mechanistic and Meaningful Activity for the Potential Treatment of DM1<br \/>\n<br \/><b><span class=\"bwuline\">Poster Number:<\/span><\/b> #P48<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Ashling Holland, PhD, Director, Research &amp; Preclinical Development\n<\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Favorable Pharmacology and Safety Profiles for the Treatment of DMD<br \/>\n<br \/><b><span class=\"bwuline\">Poster Number:<\/span><\/b> #P49<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Ashling Holland, PhD, Director, Research &amp; Preclinical Development\n<\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)<br \/>\n<br \/><b><span class=\"bwuline\">Poster Number:<\/span><\/b> #O45<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Jennifer Shoskes, PharmD, Associate Director, Clinical Development\n<\/p>\n<p><b><span class=\"bwuline\">Title:<\/span><\/b> CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping<br \/>\n<br \/><b><span class=\"bwuline\">Poster Number:<\/span><\/b> #O74<br \/>\n<br \/><b><span class=\"bwuline\">Presenter:<\/span><\/b> Bassem Morcos, MD, Medical Director, Clinical Development\n<\/p>\n<p>\nFollowing the conference, the presentations presented at the MDA Clinical &amp; Scientific Conference will be available on the Investors page of PepGen\u2019s website under Scientific Publications.\n<\/p>\n<p><b>About PGN-EDODM1<\/b><\/p>\n<p>\nPGN-EDODM1, PepGen&#8217;s investigational candidate in development for the treatment of DM1, utilizes the Company&#8217;s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the <i>DMPK<\/i> transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the <i>DMPK <\/i>transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. We believe this mechanism will allow the <i>DMPK <\/i>transcripts to continue performing its normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. We believe that this innovative therapeutic approach has considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the <i>DMPK <\/i>transcripts. The FDA has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.\n<\/p>\n<p><b>About PGN-EDO51<\/b><\/p>\n<p>\nPGN-EDO51, PepGen&#8217;s investigational candidate in development for the treatment of DMD, utilizes the Company&#8217;s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The FDA has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach.\n<\/p>\n<p><b>About PepGen<\/b><\/p>\n<p>\nPepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen\u2019s EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.\n<\/p>\n<p>\nFor more information, please visit <a rel=\"nofollow\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Fwww.pepgen.com%2F&amp;esheet=54221279&amp;newsitemid=20250310990282&amp;lan=en-US&amp;anchor=PepGen.com&amp;index=2&amp;md5=7ed2e2740aef6fe0016a5699c59bccd4\">PepGen.com<\/a>. Follow PepGen on <a rel=\"nofollow\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Fwww.linkedin.com%2Fcompany%2F33201532%2Fadmin%2Fdashboard%2F&amp;esheet=54221279&amp;newsitemid=20250310990282&amp;lan=en-US&amp;anchor=LinkedIn&amp;index=3&amp;md5=d14a28822557e2cf6f82b72ecf51065f\">LinkedIn<\/a> and <a rel=\"nofollow\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Fx.com%2FPepGenTx&amp;esheet=54221279&amp;newsitemid=20250310990282&amp;lan=en-US&amp;anchor=X&amp;index=4&amp;md5=2818ef6c9fecc8c9a203ecaff8fd53b2\">X<\/a>.\n<\/p>\n<p><img decoding=\"async\" alt=\"\" src=\"https:\/\/cts.businesswire.com\/ct\/CT?id=bwnews&amp;sty=20250310990282r1&amp;sid=flmnd&amp;distro=nx&amp;lang=en\" style=\"width:0;height:0\" \/><span class=\"bwct31415\" \/><\/p>\n<p id=\"mmgallerylink\"><span id=\"mmgallerylink-phrase\">View source version on businesswire.com: <\/span><span id=\"mmgallerylink-link\"><a href=\"https:\/\/www.businesswire.com\/news\/home\/20250310990282\/en\/\" rel=\"nofollow\">https:\/\/www.businesswire.com\/news\/home\/20250310990282\/en\/<\/a><\/span><\/p>\n<p><b><span class=\"bwuline\">Investor<br \/>\n<\/span><\/b><br \/>Dave Borah, CFA<br \/>\n<br \/>SVP, Investor Relations and Corporate Communications<br \/>\n<br \/><a rel=\"nofollow\" href=\"mailto:dborah@pepgen.com\">dborah@pepgen.com<\/a><\/p>\n<p><b><span class=\"bwuline\">Media<br \/>\n<\/span><\/b><br \/>Julia Deutsch<br \/>\n<br \/>Lyra Strategic Advisory<br \/>\n<br \/><a rel=\"nofollow\" href=\"mailto:Jdeutsch@lyraadvisory.com\">Jdeutsch@lyraadvisory.com<\/a><\/p>\n<p><b>KEYWORDS:<\/b> United States North America Texas Massachusetts<\/p>\n<p><b>INDUSTRY KEYWORDS:<\/b> Biotechnology Health Neurology Clinical Trials<\/p>\n<p><b>MEDIA:<\/b><\/p>\n<table cellpadding=\"3\" cellspacing=\"3\">\n<tr>\n<td><font face=\"Arial\" size=\"2\"><b>Logo<\/b><\/font><\/td>\n<\/tr>\n<tr>\n<td><img decoding=\"async\" src=\"https:\/\/mms.businesswire.com\/media\/20250310990282\/en\/2155755\/3\/PepGen_Logo_RGB_FullColor.jpg\" alt=\"Logo\" \/><\/td>\n<\/tr>\n<tr>\n<td><font face=\"Arial\" size=\"2\"><\/font><\/td>\n<\/tr>\n<\/table>\n","protected":false},"excerpt":{"rendered":"<p>PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference BOSTON&#8211;(BUSINESS WIRE)&#8211; PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently announced. Oral Presentations Title: CONNECT1-EDO51: A 12-Week Open-Label Phase &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference&#8221;<\/span><\/a><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-823783","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference - Market Newsdesk<\/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:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference BOSTON&#8211;(BUSINESS WIRE)&#8211; PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently announced. 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(Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently announced. Oral Presentations Title: CONNECT1-EDO51: A 12-Week Open-Label Phase &hellip; Continue reading \"PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; Scientific Conference\"","og_url":"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/","og_site_name":"Market Newsdesk","article_published_time":"2025-03-10T20:08:05+00:00","og_image":[{"url":"https:\/\/cts.businesswire.com\/ct\/CT?id=bwnews&amp;sty=20250310990282r1&amp;sid=flmnd&amp;distro=nx&amp;lang=en","type":"","width":"","height":""}],"author":"Newsdesk","twitter_card":"summary_large_image","twitter_misc":{"Written by":"Newsdesk","Est. reading time":"4 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/#article","isPartOf":{"@id":"https:\/\/www.marketnewsdesk.com\/index.php\/pepgen-announces-presentations-at-the-2025-muscular-dystrophy-association-clinical-scientific-conference\/"},"author":{"name":"Newsdesk","@id":"https:\/\/www.marketnewsdesk.com\/#\/schema\/person\/482f27a394d4fda80ecb5499e519d979"},"headline":"PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical &amp; 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