{"id":15419,"date":"2024-05-17T07:00:00","date_gmt":"2024-05-17T11:00:00","guid":{"rendered":"https:\/\/www.ptcbio.com\/?p=15419"},"modified":"2025-07-23T08:56:04","modified_gmt":"2025-07-23T12:56:04","slug":"what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa","status":"publish","type":"post","link":"https:\/\/www.ptcbio.com\/posts\/what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa\/","title":{"rendered":"What You Need to Know About Ferroptosis &amp; Friedreich&#8217;s Ataxia (FA)"},"content":{"rendered":"\n<p class=\"has-text-align-center core-block core-paragraph\"><strong><em>An Informative Q&amp;A with Jonathan Cherry, Global Project Leader <\/em><\/strong><strong><\/strong><\/p>\n\n\n\n<p class=\"core-block core-paragraph\">Our research efforts at PTC are focused on two scientific platforms \u2013 splicing and inflammation and ferroptosis \u2013 where PTC has unique expertise to discover and advance innovative therapies to the clinic.<\/p>\n\n\n\n<figure class=\"wp-block-image alignleft size-large is-resized is-style-lightbox\"><img loading=\"lazy\" decoding=\"async\" width=\"763\" height=\"1024\" src=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-763x1024.jpg\" alt=\"Image of Jonathan Cherry, PTC's Global Project Leader\" class=\"wp-image-15420\" style=\"width:188px;height:auto\" srcset=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-763x1024.jpg 763w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-223x300.jpg 223w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-768x1031.jpg 768w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-1144x1536.jpg 1144w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-1526x2048.jpg 1526w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-400x537.jpg 400w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-scaled.jpg 1907w\" sizes=\"auto, (max-width: 763px) 100vw, 763px\" \/><button class=\"modal-toggle\" type=\"button\" data-bs-toggle=\"modal\" data-bs-target=\"#mg-image-modal\" aria-label=\"View image in lightbox\"><img fetchpriority=\"high\" decoding=\"async\" width=\"1907\" height=\"2560\" src=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-scaled.jpg\" class=\"attachment-full size-full\" alt=\"Jonathan Cherry\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-scaled.jpg 1907w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-223x300.jpg 223w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-763x1024.jpg 763w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-768x1031.jpg 768w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-1144x1536.jpg 1144w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-1526x2048.jpg 1526w, https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/jonathanCherry-400x537.jpg 400w\" sizes=\"(max-width: 1907px) 100vw, 1907px\" \/><\/button><\/figure>\n\n\n\n<p class=\"core-block core-paragraph\">We recently sat down with <strong>Jonathan Cherry, Global Project Leader<\/strong>, to learn more about ferroptosis and how it relates to <a href=\"https:\/\/www.ptcbio.com\/therapeutic-areas\/friedreich-ataxia\/\">Friedreich&#8217;s ataxia (FA)<\/a>.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>How is ferroptosis involved in FA?<\/strong><\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>Jonathan: <\/strong>FA is a rare,&nbsp;genetic, progressive neuromuscular&nbsp;disease that mainly affects the&nbsp;nervous systems causing muscle weakness and loss of balance and coordination. It is usually caused by the loss of a protein called frataxin which is involved in iron metabolism and essential for proper function of mitochondria, the powerhouse of all cells in the body.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">When the mitochondria are not working correctly, cells become stressed and there is an accumulation of iron and reactive oxygen species.&nbsp;Together these initiate a feedforward cycle of inflammation that eventually triggers ferroptosis which causes the stressed cells to die. In FA, this results in the loss of nerves and muscle.&nbsp; &nbsp;<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>What investigational treatments are in clinical development that originated from<\/strong> <strong>this platform?<\/strong>&nbsp;<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>Jonathan: <\/strong>We have completed MOVE-FA, the phase 3 study of vatiquinone, an investigational treatment for FA. While the trial did not meet its primary endpoint, vatiquinone did demonstrate significant benefit on key disease subscales, including the upright stability subscale of mFARS, which is predictive of time to loss of ambulation, one of the key aspects of disease morbidity in FA patients. We plan to submit an NDA with data from the MOVE-FA study as well as data from a long-term extension study  in late 2024.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">Vatiquinone is intended to break the feedforward cycle of inflammation, prevent ferroptosis, prevent the death of nerves and muscle cells and preserve their function, slowing disease progression in FA.<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>What is the moment you\u2019re most proud of when reflecting on the development journey of these programs? <\/strong>&nbsp;<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>Jonathan: <\/strong>As leader of the vatiquinone team, I have had the privilege to represent PTC with the patients.&nbsp;Working to bring treatment to patients with rare disease is rewarding enough, but it really hits home when you are working to develop treatments for children with no other options. I hope that we can succeed and make a real difference in people\u2019s lives.&nbsp;<\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>Can you tell us about the teams working on these programs?<\/strong><\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>Jonathan: <\/strong>The vatiquinone team has over 20 members throughout the U.S., Europe, and Japan. In addition, we are working closely with health care providers at 14 clinical care sites and in 9 countries (U.S., Australia, Brazil, Canada, France, Germany, Italy, Spain and New Zealand). &nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>An Informative Q&amp;A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms \u2013 splicing and inflammation and ferroptosis \u2013 where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jonathan Cherry, Global Project Leader, to learn more about&hellip;<\/p>\n","protected":false},"author":2,"featured_media":15422,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[11],"tags":[],"media-type":[5],"therapeutic-area":[23],"class_list":["post-15419","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>What You Need to Know About Ferroptosis &amp; Friedreich&#039;s Ataxia (FA) - PTC Therapeutics (United States)<\/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.ptcbio.com\/posts\/what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"What You Need to Know About Ferroptosis &amp; Friedreich&#039;s Ataxia (FA)\" \/>\n<meta property=\"og:description\" content=\"An Informative Q&amp;A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms \u2013 splicing and inflammation and ferroptosis \u2013 where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jonathan Cherry, Global Project Leader, to learn more about&hellip;\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.ptcbio.com\/posts\/what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa\/\" \/>\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=\"2024-05-17T11:00:00+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-07-23T12:56:04+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.ptcbio.com\/wp-content\/uploads\/sites\/2\/2024\/04\/ferroptosis-QA-2-.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\\\/what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/posts\\\/what-you-need-to-know-about-ferroptosis-friedreich-ataxia-fa\\\/\"},\"author\":{\"name\":\"jacy\",\"@id\":\"https:\\\/\\\/www.ptcbio.com\\\/#\\\/schema\\\/person\\\/57224bb5c2cd0a7886dfd1c249bd5b74\"},\"headline\":\"What You Need to Know About Ferroptosis &amp; 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