/ en Thu, 16 Apr 26 14:35:55 -0400 /news/clinical-trial-prion-disease-drug-candidate-begins-enrolling-participants <span class="field field--name-title field--type-string field--label-hidden"><h1>Clinical trial of a prion disease drug candidate begins enrolling participants</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Jessica Colarossi</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-04-16T14:35:55-04:00" class="datetime">April 16, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Clinical trial of a prion disease drug candidate begins enrolling participants</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>It’s the first time the potential treatment, a small interfering RNA targeting the prion protein, is being tested in humans. </p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Jessica Colarossi </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-04-16T14:35:55-04:00" title="Thursday, April 16, 2026 - 14:35" class="datetime">April 16, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture role="img" aria-label="Sonia Vallabh and Eric Minikel in their ӳ����ý lab. "> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=i-aCTsQt 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=i-aCTsQt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=EaGols0y 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=OWuoBJgk 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=FNBE6Mbf 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=lQfv9bb6 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/DSC_7778_mo-BROAD00263923.jpg?itok=lQfv9bb6" alt="Sonia Vallabh and Eric Minikel in their ӳ����ý lab. " title="Sonia Vallabh and Eric Minikel in their ӳ����ý lab. " typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Maria Nemchuk </div> <div class="media-caption__description"> Sonia Vallabh and Eric Minikel, codirectors of ӳ����ý’s Prion Therapeutic Science program. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/2456/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/2456/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/2456/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e3ba4f3bfd0ffe62d9c277f59bc7320c8">The phase 1 trial will enroll 15 patients diagnosed with prion disease who have symptoms of the neurodegenerative disorder.</li> <li aria-level="1" data-list-item-id="e4012dd93683d9d554e4ef5e1f1a9d45c">The drug candidate is a divalent small interfering RNA (siRNA) molecule designed to cut target RNA, so cells produce less of the disease-causing prion protein. </li> <li aria-level="1" data-list-item-id="e84658b4e333dae057f52d6a6dd92e4eb">The trial will test the safety and dosing of the siRNA, and help determine whether the drug candidate should advance to larger clinical trials. </li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A new drug candidate designed to slow the progression of prion disease is entering a phase 1 <a target="_blank" href="https://clinicaltrials.gov/study/NCT07444580">clinical trial</a>, which will evaluate the potential medicine for safety and tolerability.</p> <p>Prion diseases are a class of neurodegenerative disorders that are caused by the accumulation of misfolded prion protein (PrP) in the brain. There are currently no cures, and the disease is fatal within months or years after symptoms begin. The new drug candidate, developed by ӳ����ý and University of Massachusetts Chan Medical School scientists, is a small interfering RNA (siRNA) that binds and snips RNA molecules encoding the prion protein, reducing the amount of the disease-causing protein in the brain. Previous research in animals has shown that lowering prion protein levels can delay onset and slow down the disease. The trial, called PrP-targeting siRNA Safety & Mechanism Study (PRiSM), is enrolling patients exhibiting symptoms of the disease.</p> <p>“To finally advance this drug to a human trial is the long-overdue achievement of a longstanding dream, but it's also the very beginning of learning about this drug's safety and activity in humans,” said <a target="_blank" href="/bios/eric-minikel">Eric Minikel</a>, principal investigator of the trial and codirector of ӳ����ý’s Prion Therapeutic Science program. “I am looking forward to finding out whether this candidate has a future as a drug in our disease, but no matter what the outcome, as sponsor-investigators, we will learn a lot about how to run a clinical trial in prion disease, and we plan to broadly and publicly share our data and findings to benefit all sponsors who want to develop drugs for prion disease.” </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--image-text paragraph--view-mode--left"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-pt-image-text-left-right-"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture role="img" aria-label="Vial of the divalent siRNA drug candidate"> <source srcset="/files/styles/pt_image_text_left_right_laptop/public/2026-04/siRnavial.png?itok=4bLS07pL 1x" media="all and (min-width: 1921px)" type="image/png" width="640" height="500"/> <source srcset="/files/styles/pt_image_text_left_right_laptop/public/2026-04/siRnavial.png?itok=4bLS07pL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="640" height="500"/> <source srcset="/files/styles/pt_image_text_left_right_laptop/public/2026-04/siRnavial.png?itok=4bLS07pL 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="640" height="500"/> <source srcset="/files/styles/pt_image_text_left_right_laptop/public/2026-04/siRnavial.png?itok=4bLS07pL 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="640" height="500"/> <source srcset="/files/styles/pt_image_text_left_right_phone/public/2026-04/siRnavial.png?itok=ufU1nYZi 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="500" height="391"/> <source srcset="/files/styles/pt_image_text_left_right_phone/public/2026-04/siRnavial.png?itok=ufU1nYZi 1x" media="all and (max-width: 539px)" type="image/png" width="500" height="391"/> <img loading="lazy" width="500" height="391" src="/files/styles/pt_image_text_left_right_phone/public/2026-04/siRnavial.png?itok=ufU1nYZi" alt="Vial of the divalent siRNA drug candidate" title="Vial of the divalent siRNA drug candidate" typeof="foaf:Image" /> </picture> <div class="media-caption">A vial of the divalent siRNA drug candidate.</div> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Argonaut Manufacturing Services </div> <div class="media-caption__description"> A vial of the divalent siRNA drug candidate. </div> </div> </article> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>The drug candidate is a divalent siRNA: two identical siRNAs that are linked together and designed to distribute more broadly in the brain than single siRNAs. The divalent siRNA was developed at the UMass Chan Medical School by <a href="https://www.umassmed.edu/khvorovalab/" target="_blank">Anastasia Khvorova</a> and her lab. Khvorova, Minikel, and <a href="/bios/sonia-vallabh" target="_blank">Sonia Vallabh</a>, codirector of ӳ����ý’s Prion Therapeutic Science program, have been working together since 2019 on developing an siRNA-based drug to target prion protein mRNA. Vallabh and Minikel, who are wife and husband, have dedicated their lives to creating a cure or treatment for prion disease, after learning in 2011 that Vallabh has the genetic mutation that causes the disease.</p> <p>In March 2025, the U.S. Food and Drug Administration (FDA) cleared the team’s Investigational New Drug (IND) application to start a clinical trial on this drug candidate — a document that is usually kept private by drug companies. However, Minikel and Vallabh <a href="https://www.cureffi.org/2025/04/14/open-ind-whats-next/" target="_blank">published their IND</a> filing publicly, and have committed to sharing new insights openly.</p> <p>This green light from the FDA — a significant step in the drug development process — follows promising animal data from the Minikel/Vallabh lab. The scientists showed that a divalent siRNA lowered prion protein in mice by 49 percent and resulted in a 64 percent increase in survival time with a single dose after symptom onset. The study is available as a <a href="https://www.biorxiv.org/content/10.1101/2024.12.05.627039v6" target="_blank">preprint</a>.</p> <p>The clinical trial is being supported by NeuroNEXT, or Network for Excellence in Neuroscience Clinical Trials, a program of the National Institute of Neurological Disorders and Stroke (NINDS), a part of the National Institutes of Health. NeuroNEXT provides both funding as well as infrastructure to run the trial, including trial sites, a clinical coordinating center housed at Mass General Hospital’s Neurology Department, and a data and statistics center at the University of Iowa.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Learn more</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/gene-editing-extends-lifespan-mouse-model-prion-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture role="img" aria-label="Erik Minikel and Sonia Vallabh stand side-by-side in a lab, wearing lab coats."> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=xHGOOyNo 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=xHGOOyNo 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=a6YwAq3t 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=a6YwAq3t 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=uO93ZKD1 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=PO6-CXkf 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"> <a href="/news/gene-editing-extends-lifespan-mouse-model-prion-disease"><img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_DSC_7699_mo-BROAD00263909.jpg?itok=PO6-CXkf" alt="Erik Minikel and Sonia Vallabh stand side-by-side in a lab, wearing lab coats." title="Erik Minikel and Sonia Vallabh stand side-by-side in a lab, wearing lab coats." typeof="foaf:Image"></a> </picture> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-extends-lifespan-mouse-model-prion-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing extends lifespan in mouse model of prion disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/therapy-candidate-fatal-prion-diseases-turns-disease-causing-gene" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture role="img" aria-label="Images of a mouse brain show many yellow dots in the left panel, representing neurons expressing the prion protein. The right panel shows most of those neurons as purple dots, meaning that they no longer expressing the protein after being treated with the CHARM gene-silencing technology."> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=xtDey6CM 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=xtDey6CM 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=6On8gkpP 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=6On8gkpP 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=YcfnHoL0 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=GGKs_BRA 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"> <a href="/news/therapy-candidate-fatal-prion-diseases-turns-disease-causing-gene"><img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/WeissmanVallabh_graphic-01.png?h=e8abbf60&itok=GGKs_BRA" alt="Images of a mouse brain show many yellow dots in the left panel, representing neurons expressing the prion protein. The right panel shows most of those neurons as purple dots, meaning that they no longer expressing the protein after being treated with the CHARM gene-silencing technology." title="Images of a mouse brain show many yellow dots in the left panel, representing neurons expressing the prion protein. The right panel shows most of those neurons as purple dots, meaning that they no longer expressing the protein after being treated with the CHARM gene-silencing technology." typeof="foaf:Image"></a> </picture> </div> </article> </div> <div class="node__content"> <a href="/news/therapy-candidate-fatal-prion-diseases-turns-disease-causing-gene" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">A therapy candidate for fatal prion diseases turns off disease-causing gene</span> </a> </div> </article> </div> <div class="field__item"><article about="/blog/registry-prion-diseases-comes-life" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture role="img" aria-label> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=8f3rvjdv 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=8f3rvjdv 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=uLKibWAB 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=uLKibWAB 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=i2wMgIKq 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=9-ckoYDM 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"> <a href="/blog/registry-prion-diseases-comes-life"><img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/blog/images/2017/signing-up-for-prion-registry_CureFFI_0.png?itok=9-ckoYDM" alt="Eric Minikel and Sonia Vallabh" typeof="foaf:Image"></a> </picture> </div> </article> </div> <div class="node__content"> <a href="/blog/registry-prion-diseases-comes-life" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">A registry for prion diseases comes to life</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>“Eric and Sonia are visionaries who are not only top-notch scientists, but have enormous ethical conviction. I’m honored to have an opportunity to work with them,” said Khvorova, professor in UMass’s RNA Therapeutics Institute and Program in Molecular Medicine. “Their commitment to transparency is paving a new path for running a clinical trial, so we’re not only advancing therapy for prion disease, it’s advancing the field as a whole.”</p> <p>The initial phase of the trial will enroll 15 symptomatic patients who will each receive one dose of the siRNA by lumbar puncture, and new patients enrolled over the course of the trial will receive larger doses than patients enrolled earlier. The trial will also have an observational arm consisting of 15 additional participants who will not receive any treatment. Minikel says they hope to add pre-sympomatic participants in the future. They also plan to share safety data as soon as they are able.</p> <p>“Standard drug development is, for all sorts of reasons, not well set up to be a learning system,” Vallabh said. “But we can’t afford to treat each shot on goal as a self-contained unit – everything needs to feed the mission. I wish the absolute best for this exact molecule, and I also know that however it performs in the clinic, this experience must move us forward. It’s our job to figure out how.”</p> <p>This is now the second clinical trial that Vallabh and her team at ӳ����ý have helped to enable. The first one, initiated by Ionis Pharmaceuticals, began in 2023 for drug candidate <a target="_blank" href="https://clinicaltrials.gov/study/NCT06153966">ION717</a> and is ongoing. In their lab, Minikel and Vallabh are pursuing other possible therapeutic approaches for prion disease, including <a target="_blank" href="/news/therapy-candidate-fatal-prion-diseases-turns-disease-causing-gene">epigenetic</a> and <a target="_blank" href="/news/gene-editing-extends-lifespan-mouse-model-prion-disease">gene editing</a>.</p> <p>To learn more about PRiSM, visit <a target="_blank" href="https://clinicaltrials.gov/study/NCT07444580?cond=Prion%20Disease&aggFilters=status:not%20rec&rank=1">ClinicalTrials.gov </a>and <a target="_blank" href="http://CureFFI.org">CureFFI.org</a>, where Minikel shares information for the prion patient and scientific communities. For patient resources, Minikel and Vallabh recommend visiting the <a target="_blank" href="https://cjdfoundation.org/">CJD Foundation</a>.  </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>The drug development was funded by National Institutes of Health grants and by donations from members of the prion disease community to the ӳ����ý and to <a href="https://www.prionalliance.org/" target="_blank">Prion Alliance</a>. This clinical trial is being funded by the National Institutes of Neurological Disorders and Stroke through the <a href="https://neuronext.org/" target="_blank">NeuroNEXT program</a> (Grant 3OT2NS138339-01S2).</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/neurodegeneration" hreflang="en">Neurodegeneration</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/clinical-trials" hreflang="en">Clinical trials</a></div> </div> </div> </div> </div> </div> Thu, 16 Apr 2026 18:35:55 +0000 jcolaros 5560386 at