{"id":15085,"date":"2023-12-18T07:00:00","date_gmt":"2023-12-18T12:00:00","guid":{"rendered":"https:\/\/www.ptcbio.com\/?p=15085"},"modified":"2023-12-12T16:09:33","modified_gmt":"2023-12-12T21:09:33","slug":"a-whole-team-in-one-molecule-ptcs-splicing-platform","status":"publish","type":"post","link":"https:\/\/www.ptcbio.com\/posts\/a-whole-team-in-one-molecule-ptcs-splicing-platform\/","title":{"rendered":"A Whole Team in One Molecule: PTC\u2019s Splicing Platform"},"content":{"rendered":"\n<p class=\"core-block core-paragraph\">There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">Here, \u201csplicing\u201d refers to PTC\u2019s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which are known as splicing modifiers, that are designed to strengthen the interaction between the cellular splicing machinery and the pre-mRNA to help make splicing more efficient.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">PTC pioneered the idea that splicing can be modulated utilizing small molecules, built on the team\u2019s expertise in RNA biology. Over the last 20 years, the PTC research team has perfected the RNA splicing technology. Using this technology, the team successfully identified an oral small molecule for the treatment of spinal muscular atrophy (SMA) \u2013 the first ever small molecule approved to specifically target splicing. The treatment targets and encourages a faulty exon in the SMN2 \u2018back-up\u2019 gene to produce a greater amount of functional SMN protein.<\/p>\n\n\n\n<div  class=\"quote-block  color-theme--white\">\n\n\t\n\t<div class=\"quote-block__content\">\n\t\t<blockquote class=\"quote-block__text color-theme-border--white\">\n\t\t\t<div class=\"quote-block__heading heading--4\">\n\t\t\t\tOur expertise in RNA biology was critical for the discovery of the SMA treatment. It was this expertise that led us to develop technology to target splicing and allowed us to move forward with this scientific platform.\t\t\t<\/div>\n\t\t<\/blockquote>\n\n\t\t\t\t\t<cite class=\"quote-block__author quote-block__author--name\">\n\t\t\t\tChris Trotta\t\t\t<\/cite>\n\t\t\n\t\t\t\t\t<cite class=\"quote-block__author quote-block__author--title\">\n\t\t\t\tSenior Vice President, Biology\t\t\t<\/cite>\n\t\t\t<\/div>\n\n<\/div>\n\n\n\n\n<p class=\"core-block core-paragraph\">The PTC research team has also applied the splicing platform to Huntington\u2019s disease (HD) for a potential treatment for HD called PTC518. HD is a rare disease caused by a defect in the <em>HTT<\/em> gene which produces a mutated HTT protein. PTC518, a small molecule that can be taken orally, induces the inclusion of a very weak exon with a premature stop codon to become part of the HTT mRNA. This splicing modification prevents the production of the full length HTT protein and causes rapid degradation of the HTT mRNA. PTC518 has demonstrated a dose dependent reduction in HTT mRNA and protein.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">\u201cThe learnings from our discovery process in SMA and HD have allowed our discovery team to make the types of hypotheses to get us to the next level,\u201d says Suresh Babu, Senior Research Fellow, Chemistry. \u201cThese important insights will help us determine how we find the next molecule.\u201d<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">PTC\u2019s approach to drug discovery, and the passionate research team employing this technology, led to the creation of these compounds.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">\u201cWe don\u2019t just take one shot when it comes to drug discovery,\u201d says Matt Woll, Senior Vice President, Chemistry. \u201cUltimately, the science will tell us the best approach, but we have to try many times to get there.\u201d<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">The discovery process and the way the platform is optimized for the treatment of rare diseases is also a product of cross-functional collaboration.<\/p>\n\n\n\n<div  class=\"quote-block  color-theme--white\">\n\n\t\n\t<div class=\"quote-block__content\">\n\t\t<blockquote class=\"quote-block__text color-theme-border--white\">\n\t\t\t<div class=\"quote-block__heading heading--4\">\n\t\t\t\tThe culmination of many efforts comes together in one molecule. We all work together toward one goal, and there is a whole team behind one molecule.\t\t\t<\/div>\n\t\t<\/blockquote>\n\n\t\t\t\t\t<cite class=\"quote-block__author quote-block__author--name\">\n\t\t\t\t Anu Bhattacharyya\t\t\t<\/cite>\n\t\t\n\t\t\t\t\t<cite class=\"quote-block__author quote-block__author--title\">\n\t\t\t\tVice President, Biology\t\t\t<\/cite>\n\t\t\t<\/div>\n\n<\/div>\n\n\n\n\n<p class=\"core-block core-paragraph\">PTC\u2019s RNA splicing technology has broad potential to identify new treatments for patients suffering from a variety of diseases. We continue to identify novel small molecules that modify splicing of disease-relevant genes, either by promoting inclusion of specific exons or by forcing an exon to be skipped. PTC scientists will continue to leverage our innovative RNA splicing platform to discover new therapies that address the underlying causes of life-limiting diseases.&nbsp;<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">\u201cWe are illuminating a new area in splicing,\u201d says Chris. \u201cWe have the experience and know-how to avoid roadblocks along the drug discovery process. We are applying all of this as we work hard to get to the next discovery of a molecule.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"<p>There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, \u201csplicing\u201d refers to PTC\u2019s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which&hellip;<\/p>\n","protected":false},"author":2,"featured_media":15077,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[11],"tags":[],"media-type":[5],"therapeutic-area":[],"class_list":["post-15085","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science-and-innovation"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.5 (Yoast SEO v27.5) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>A Whole Team in One Molecule: PTC\u2019s Splicing Platform - PTC Therapeutics (United States)<\/title>\n<meta name=\"description\" content=\"Read about the splicing platform and the passionate research team employing this technology.\" \/>\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.ptcbio.com\/posts\/a-whole-team-in-one-molecule-ptcs-splicing-platform\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"A Whole Team in One Molecule: PTC\u2019s Splicing Platform\" \/>\n<meta property=\"og:description\" content=\"Read about the splicing platform and the passionate research team employing this technology.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.ptcbio.com\/posts\/a-whole-team-in-one-molecule-ptcs-splicing-platform\/\" \/>\n<meta property=\"og:site_name\" content=\"PTC Therapeutics (United States)\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/PTCTherapeutics\" \/>\n<meta property=\"article:published_time\" content=\"2023-12-18T12:00:00+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2023\/12\/splicing-newsroom.png\" \/>\n\t<meta property=\"og:image:width\" content=\"466\" \/>\n\t<meta property=\"og:image:height\" content=\"262\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"jacy\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@PTCBio\" \/>\n<meta name=\"twitter:site\" content=\"@PTCBio\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"jacy\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/posts\\\/a-whole-team-in-one-molecule-ptcs-splicing-platform\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/posts\\\/a-whole-team-in-one-molecule-ptcs-splicing-platform\\\/\"},\"author\":{\"name\":\"jacy\",\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/#\\\/schema\\\/person\\\/57224bb5c2cd0a7886dfd1c249bd5b74\"},\"headline\":\"A Whole Team in One Molecule: PTC\u2019s Splicing Platform\",\"datePublished\":\"2023-12-18T12:00:00+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/posts\\\/a-whole-team-in-one-molecule-ptcs-splicing-platform\\\/\"},\"wordCount\":527,\"publisher\":{\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/posts\\\/a-whole-team-in-one-molecule-ptcs-splicing-platform\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.ptcbio.com\\\/wp-content\\\/uploads\\\/sites\\\/2\\\/2023\\\/12\\\/splicing-newsroom.png\",\"articleSection\":[\"Science &amp; 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