{"id":3110,"date":"2025-03-08T13:21:47","date_gmt":"2025-03-08T10:21:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3110"},"modified":"2025-03-08T13:21:49","modified_gmt":"2025-03-08T10:21:49","slug":"faq-solid-biosciences-sgt-003-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/faq-solid-biosciences-sgt-003-gene-therapy\/","title":{"rendered":"Solid Biosciences SGT-003 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Many research companies around the world are researching treatments for Duchenne, one of the most devastating muscle diseases. One of these is Solid Biosciences&#8217; SGT-003 gene therapy, which is currently in development and uses new generation technologies. In this article, we will explore frequently asked questions about Solid Biosciences&#8217; SGT-003 gene therapy.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Things to Know About SGT-003 Gene Therapy<\/h2><nav><ul><li class=\"\"><a href=\"#what-is-sgt-003-gene-therapy\">What is SGT-003 Gene Therapy?<\/a><\/li><li class=\"\"><a href=\"#who-is-eligible-for-sgt-003-gene-therapy\">Who is Eligible for SGT-003 Gene Therapy?<\/a><\/li><li class=\"\"><a href=\"#who-is-not-eligible-for-sgt-003-gene-therapy\">Who is Not Eligible for SGT-003 Gene Therapy?<\/a><\/li><li class=\"\"><a href=\"#interim-clinical-trial-results-of-sgt-003-gene-therapy\">Interim Clinical Trial Results of SGT-003 Gene Therapy<\/a><\/li><li class=\"\"><a href=\"#sgt-003-and-elevidys-comparison\">SGT-003 and Elevidys Comparison<\/a><\/li><li class=\"\"><a href=\"#price-of-sgt-003-gene-therapy\">Price of SGT-003 Gene Therapy<\/a><\/li><li class=\"\"><a href=\"#conclusion\">Conclusion<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-sgt-003-gene-therapy\">What is SGT-003 Gene Therapy?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">AAV-SLB101, a proprietary, next-generation capsid that was logically created to target integrin receptors, and a differentiated microdystrophin construct are both components of SGT-003, an investigational gene therapy that has demonstrated improved transduction in the heart and skeletal muscles while reducing liver targeting in nonclinical studies.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">According to these design elements, SGT-003 may be the finest experimental gene therapy available for treating Duchenne.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-is-eligible-for-sgt-003-gene-therapy\">Who is Eligible for SGT-003 Gene Therapy?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">According to the clinical trial launched by Solid Biosciences in May 2024, participants are selected between the ages of 4 and 11. [<a href=\"https:\/\/www.clinicaltrials.gov\/study\/NCT06138639\" target=\"_blank\" rel=\"noreferrer noopener\">NCT06138639<\/a>]<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">When considering the Inclusion Criteria, the following situations are taken into consideration for the participants:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Participants who are ambulatory. Ambulatory as defined as &#8220;being able to walk without the use of an assistive device.&#8221;<\/li>\n\n\n\n<li>Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing.<\/li>\n\n\n\n<li>Negative for AAV antibodies.<\/li>\n\n\n\n<li>On a stable dose of at least 0.5 mg\/kg\/day of oral daily prednisone or 0.75 mg\/kg\/day deflazacort for \u226512 weeks prior to entering the study.<\/li>\n\n\n\n<li>Meet 10-meter walk\/run time criteria<\/li>\n\n\n\n<li>Meet time to rise from supine criteria<\/li>\n\n\n\n<li>Participant has body weight: \u226450 kg<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-is-not-eligible-for-sgt-003-gene-therapy\">Who is Not Eligible for SGT-003 Gene Therapy?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Considering the exclusion criteria, it is stated that patients with some exon deletions cannot participate in this study.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">This may mean that Solid Biosciences SGT-003 gene therapy cannot be used for these exon deletions.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"interim-clinical-trial-results-of-sgt-003-gene-therapy\">Interim Clinical Trial Results of SGT-003 Gene Therapy<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Biosciences announced interim results for its SGT-003 gene therapy on February 18, 2025. [<a href=\"https:\/\/dmdwarrior.com\/solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003\/\" target=\"_blank\" rel=\"noreferrer noopener\">Read More<\/a>]<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Mean vector copies per nucleus: 18.7 (N=3),<\/li>\n\n\n\n<li>Mean microdystrophin expression: 110% (N=3), as measured by western blot,<\/li>\n\n\n\n<li>Mean microdystrophin expression: 108% (N=3), as measured by mass spectrometry,<\/li>\n\n\n\n<li>Mean percent dystrophin positive fibers: 78% (N=3), as measured by immunofluorescence,<\/li>\n\n\n\n<li>Mean beta sarcoglycan percent positive fibers: 70% (N=3),<\/li>\n\n\n\n<li>Mean nNOS (neuronal nitric oxide synthase) percent positive fibers: 42% (N=3),<\/li>\n\n\n\n<li>Improvements in 7 additional muscle integrity biomarkers (N=3), and<\/li>\n\n\n\n<li>Early mean improvement in left ventricular ejection fraction (LVEF) of 8% from baseline at Day 180 (N=2).<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">In mid-2025, the Company plans to request a meeting with the FDA to discuss potential accelerated approval pathways for SGT-003. [<a href=\"https:\/\/dmdwarrior.com\/solid-biosciences-plans-meeting-with-fda-in-mid-2025-for-sgt-003-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Read More<\/a>]<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"sgt-003-and-elevidys-comparison\">SGT-003 and Elevidys Comparison<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Promising interim trial results of the SGT-003 gene therapy have drawn attention to this research, and patients and their families have begun to research whether SGT-003 or Elevidys is better.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">When clinical data are examined, it can be seen that SGT-003 has higher microdystrophin expression. [<a href=\"https:\/\/investors.solidbio.com\/news-releases\/news-release-details\/solid-biosciences-reports-fourth-quarter-and-full-year-2024\" target=\"_blank\" rel=\"noreferrer noopener\">Read More<\/a>]<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"price-of-sgt-003-gene-therapy\">Price of SGT-003 Gene Therapy<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Since SGT-003 is still in the research phase, Solid Biosciences has not yet announced the price of the gene therapy.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Hopefully SGT-003 will work, gain approval, and be available on the pharmaceutical market at a price that every family can afford.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">Conclusion<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Many research companies around the world continue their work to treat Duchenne muscular dystrophy. New generation gene therapies are still spreading hope for patients. Solid Biosciences&#8217; SGT-003 gene therapy could rival Elevidys and pave the way for easier access for patients in terms of cost.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Learn More: <a href=\"https:\/\/dmdwarrior.com\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\">Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Many research companies around the world are researching treatments for Duchenne, one of the most devastating muscle diseases. One of these is Solid Biosciences&#8217; SGT-003 gene therapy, which is currently in development and uses new generation technologies. In this article, we will explore frequently asked questions about Solid Biosciences&#8217; SGT-003 gene therapy. What is SGT-003 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3134,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[110,170,109],"class_list":["post-3110","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3110","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=3110"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3110\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/3134"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=3110"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=3110"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=3110"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}