University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome

<!doctype html><html lang="en"><head> <meta charset="utf-8"> <title>University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome</title> <link rel="shortcut icon" href="https://oxfordglobal.com/hubfs/OXFORD_GLOBAL-LOGO_SYMBOL.jpg"> <meta name="description" content="The collaboration hopes to develop a AAV gene therapy for rare genetic disease Angelman syndrome."> <meta name="viewport" content="width=device-width, initial-scale=1"> <meta property="og:description" content="The collaboration hopes to develop a AAV gene therapy for rare genetic disease Angelman syndrome."> <meta property="og:title" content="University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome"> <meta name="twitter:description" content="The collaboration hopes to develop a AAV gene therapy for rare genetic disease Angelman syndrome."> <meta name="twitter:title" content="University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome"> <style> a.cta_button{-moz-box-sizing:content-box !important;-webkit-box-sizing:content-box !important;box-sizing:content-box !important;vertical-align:middle}.hs-breadcrumb-menu{list-style-type:none;margin:0px 0px 0px 0px;padding:0px 0px 0px 0px}.hs-breadcrumb-menu-item{float:left;padding:10px 0px 10px 10px}.hs-breadcrumb-menu-divider:before{content:'›';padding-left:10px}.hs-featured-image-link{border:0}.hs-featured-image{float:right;margin:0 0 20px 20px;max-width:50%}@media (max-width: 568px){.hs-featured-image{float:none;margin:0;width:100%;max-width:100%}}.hs-screen-reader-text{clip:rect(1px, 1px, 1px, 1px);height:1px;overflow:hidden;position:absolute !important;width:1px} </style> <link rel="stylesheet" href="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/135726131582/1732113079147/git/og-stage/css/main.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/fontawesome.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/brands.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/solid.min.css">  <style id="hs_editor_style" type="text/css"> #hs_cos_wrapper_module_17019620504109 { display: block !important; margin-bottom: 0px !important } </style>  <script> var _hsp = window._hsp = window._hsp || []; window.dataLayer = window.dataLayer || []; function gtag(){dataLayer.push(arguments);} var useGoogleConsentModeV2 = true; var waitForUpdateMillis = 1000; if (!window._hsGoogleConsentRunOnce) { window._hsGoogleConsentRunOnce = true; gtag('consent', 'default', { 'ad_storage': 'denied', 'analytics_storage': 'denied', 'ad_user_data': 'denied', 'ad_personalization': 'denied', 'wait_for_update': waitForUpdateMillis }); if (useGoogleConsentModeV2) { _hsp.push(['useGoogleConsentModeV2']) } else { _hsp.push(['addPrivacyConsentListener', function(consent){ var hasAnalyticsConsent = consent && (consent.allowed || (consent.categories && consent.categories.analytics)); var hasAdsConsent = consent && (consent.allowed || (consent.categories && consent.categories.advertisement)); gtag('consent', 'update', { 'ad_storage': hasAdsConsent ? 'granted' : 'denied', 'analytics_storage': hasAnalyticsConsent ? 'granted' : 'denied', 'ad_user_data': hasAdsConsent ? 'granted' : 'denied', 'ad_personalization': hasAdsConsent ? 'granted' : 'denied' }); }]); } } gtag('js', new Date()); gtag('set', 'developer_id.dZTQ1Zm', true); gtag('config', 'G-43QK8SYTST'); </script> <script async src="https://www.googletagmanager.com/gtag/js?id=G-43QK8SYTST"></script>  <link rel="canonical" href="https://oxfordglobal.com/discovery-development/resources/university-of-pennsylvania-and-fast-to-develop-aav-gene-therapy-for-angelman-syndrome"> <meta property="og:image" content="https://oxfordglobal.com/hubfs/University%20of%20Pennsylvania%20and%20FAST%20to%20Develop%20AAV%20Gene%20Therapy%20for%20Angelman%20Syndrome.jpg"> <meta property="og:image:width" content="1600"> <meta property="og:image:height" content="900"> <meta name="twitter:image" content="https://oxfordglobal.com/hubfs/University%20of%20Pennsylvania%20and%20FAST%20to%20Develop%20AAV%20Gene%20Therapy%20for%20Angelman%20Syndrome.jpg"> <meta property="og:url" content="https://oxfordglobal.com/discovery-development/resources/university-of-pennsylvania-and-fast-to-develop-aav-gene-therapy-for-angelman-syndrome"> <meta name="twitter:card" content="summary"> <meta http-equiv="content-language" content="en"> <meta name="generator" content="HubSpot"></head> <body class="discovery-development"> <div class="body-wrapper hs-content-id-177028096592 hs-site-page page "> <div data-global-resource-path="git/og-stage/templates/partials/header.html"> <header class="website-header "> <div class="navbar"> <div class="navbar-brands"> <a href="/discovery-development" class="logo-link current-brand"> <img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/Discovery%20%26%20Development.svg" class="logo-image"> <i class="dropdown fa-solid fa-chevron-down"></i> </a> <div class="brands"> <div class="intro"> <h2> Explore our brands </h2> <div class="desc"> Our brands bring together the industry's most influential leaders and scientific experts, inspiring thought-provoking and insightful content that offers a unique perspective on R&D trends and challenges. </div> </div> <div class="items"> <div class="item primary-brand"> <a href="/" class="logo-link"><img src="https://oxfordglobal.com/hubfs/logo.jpg" class="logo-image"></a> </div> <div class="item"> <a href="/precision-medicine" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/Precision%20Medicine.svg" class="logo-image"></a> </div> <div class="item active-item"> <a href="/discovery-development" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/Discovery%20%26%20Development.svg" class="logo-image"></a> </div> <div class="item"> <a href="/nextgen-biomed" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/NextGen%20Biomed.svg" class="logo-image"></a> </div> </div> </div> </div> <div class="flex flex-align-center navbar-nav"> <a href="#" class="navbar-nav-toggle"> <div class="toggle-icons"> <i class="fa-solid fa-bars toggle-open"></i> <i class="fa-solid fa-xmark toggle-close"></i> </div> </a> <ul class="navbar-nav-items"> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Events <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Events.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex-3 divider g-20"> <div class="subnav-item-content bottom-mp-fix"> <h3> In-Person & Online Events </h3> <p>Pivotal moments where high-level industry experts and thought leaders come together for focused knowledge sharing and unparalleled networking opportunities.</p> </div> <div class="subnav-item"> <div class="event-pills"> <a href="/discovery-development/events/discovery-development-europe-2025" class="pill center"> <div class="b">Discovery & Development Europe 2025</div> <div class="heading-smallcaps"> 01 - 02 Jul 2025 | Basel, Switzerland </div> </a> <a href="/discovery-development/events/discovery-development-us-2025" class="pill center"> <div class="b">Discovery & Development US 2025</div> <div class="heading-smallcaps"> 02 - 03 Oct 2025 | San Diego, CA </div> </a> </div> </div> </div> <div class="subnav-item flex-2"> <div class="subnav-item-content bottom-mp-fix"> <h3> Online Activities </h3> <p>Our Monthly Science Exchanges and Webinars keep you connected to the conversation.</p> </div> <div class="online-activities-list flex flex-column g-10">  <div class="online-activity"> <a href="/discovery-development/online#Controlled%20Release%20Formulations" class="cta" target="_blank"> <h3 class="other-resources">26/06/24 - Controlled Release Formulations</h3> </a> </div> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/online" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> All Online Activities</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Community <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Community.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> About </h3> <p>We empower scientific decision-makers from the world’s most innovative biopharma organisations to make informed pipeline decisions in early R&D, accelerating their route from target ID to novel clinical candidate.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/about" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Community </h3> <p>Our community extends across the entire early R&D ecosystem, including biopharma, solution providers, start-ups, academics, and investors.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/community" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1 "> <div class="subnav-item-content bottom-mp-fix"> <h3> Media Partners </h3> <p>We work with leading journal publications on reciprocal marketing arrangements.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/media-partners" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Services <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Services.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> In-Person Services </h3> <p>At Oxford Global, our events are designed to connect key life science figures and innovative solution providers, accelerating scientific advancement. Our goal is to create valuable connections and an impactful experience through innovative and collaborative formats.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/in-person-services" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1"> <div class="subnav-item-content bottom-mp-fix"> <h3> Digital Marketing Services </h3> <p>Oxford Global’s products offer a powerful channel to associate your brand with the latest advancements within the industry whilst educating and influencing the industry throughout their buying process.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/digital-marketing-services" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Resources <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Resources.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex-2 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Browse All Resources </h3> <p>Scientific & technical insights on the latest targets, platforms and processes impacting drug discovery & development.</p> </div> <div class="subnav-item-content-more"> <a href="/discovery-development/resources" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> All Discovery & Development Resources</a> </div> </div> <div class="subnav-item flex-4 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Latest Resources </h3> <div class="resources"> <div class="resource"> <div class="resource-cover"> <a href="/discovery-development/resources/biomedgpt-adopting-a-generalist-approach-to-biomedical-ai" class="resource-cover-link"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Resources/Discovery%20and%20Development/BiomedGPT%20Adopting%20a%20Generalist%20Approach%20to%20Biomedical%20AI%20.png" class="resource-cover-image"> </a> </div> <div class="resource-content"> <h3 class="resource-heading"> <a href="/discovery-development/resources/biomedgpt-adopting-a-generalist-approach-to-biomedical-ai">BiomedGPT: Adopting a Generalist Approach to Biomedical AI </a> </h3> <div class="resource-abstract"> <span class="resource-date">11/11/24 - </span> BiomedGPT, a versatile AI tool used for biomedical research demonstrated impressive results across a wide range of healthcare tasks. </div> </div> </div> <div class="top-divider"></div> <div class="resource"> <div class="other-resources"><a href="/discovery-development/resources/the-application-of-rnai-therapeutics-for-cardiovascular-disease-treatment">07/11/24 - The Application of RNAi Therapeutics for Cardiovascular Disease Treatment</a></div> </div> <div class="top-divider"></div> <div class="resource"> <div class="other-resources"><a href="/discovery-development/resources/ai-ml-advancements-in-drug-formulation-the-potential-of-self-driving-labs">16/10/24 - AI/ML Advancements in Drug Formulation – The Potential of Self-Driving Labs </a></div> </div> </div> </div> </div> <div class="subnav-item flex-3"> <div class="subnav-item-content bottom-mp-fix"> <h3> Featured Resource </h3> <div class="resources flex flex-column g-5"> <a href="https://oxfordglobal.com/discovery-development/resources/sponsored/case-studies/ardigen/advancing-drug-discovery-through-innovative-technologies-strategic-collaboration"> <div class="sponsor-resource flex-column"> <div class="resource-cover img-fluid"> <div class="sponsor-resource-cover-link"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Resources/Discovery/Advancing%20Drug%20Discovery%20Mock%20Up.png" class="sponsor-resource-bubble-image"> </div> </div> <div class="resource-content p-15"> <h3 class="resource-heading"> Advancing Drug Discovery Through Innovative Technologies & Strategic Collaboration </h3> <div class="resource-abstract"> A Concise Report Featuring Insights From The Prominent Thought Leaders Of Discovery & Automate 2024 </div> </div> </div> </a> </div> </div> </div> </div> </div> </li> <li class="navbar-nav-item show-sm hide-md hide-lg hide-xl"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});" class="navbar-nav-item-link">Contact</a> </li> <li class="navbar-nav-item show-sm hide-md hide-lg hide-xl"> <a href="#" class="navbar-nav-item-link">Our Brands <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-items"> <h2> Explore our brands </h2> <div class="desc"> Our brands bring together the industry's most influential leaders and scientific experts, inspiring thought-provoking and insightful content that offers a unique perspective on R&D trends and challenges. </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/">Oxford Global</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/precision-medicine">Precision Medicine</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/discovery-development">Discovery & Development</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/nextgen-biomed">NextGen Biomed</a> </div> </div> </div> </div> </li> </ul> </div> <div class="flex flex-align-center hide-sm show-md show-lg show-xl"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});" class="navbar-button">Contact</a> </div> </div> </header></div> <main id="main-content"> <div class="resource-single"> <div class="header bg-primary-light"> <div class="container-wrapper"> <div class="wide-container header-container"> <div class="header-content flex-1"> <div class="heading-smallcaps"> Industry Spotlights & Insight Articles </div> <h1 class="heading"> University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome </h1> <div class="byline"> Edited by Tom Cohen | 12 December 2023 </div> <div class="abstract"> The collaboration hopes to develop a AAV gene therapy for rare genetic disease Angelman syndrome. </div> </div> <div class="header-graphic flex-1"> <img src="https://oxfordglobal.com/hubfs/University%20of%20Pennsylvania%20and%20FAST%20to%20Develop%20AAV%20Gene%20Therapy%20for%20Angelman%20Syndrome.jpg" class="header-graphic-image"> </div> </div> </div> </div> <div class="container-wrapper"> <div class="wide-container"> <div class="content"> <div class="content-body text-gap"> <p>The University of Pennsylvania has entered into an exclusive partnership with The Foundation for Angelman Syndrome Therapeutics (FAST) to develop an investigational gene therapy to treat Angelman syndrome (AS).</p> <p>Collaboration between FAST and UPenn goes back as far as 2017. Since then, the foundation has funded preclinical research at the university’s<span> </span><a href="https://gtp.med.upenn.edu/" target="_blank" rel="noreferrer noopener">Gene Therapy Program</a><span> </span>to develop adeno-associated virus (AAV) AS gene therapies targeting the central nervous system (CNS).</p> <p>“Our collaboration with the team at FAST, and the entire Angelman syndrome community, has been incredibly productive and rewarding, since its inception,” said Jim Wilson, director of the Gene Therapy Program at the University of Pennsylvania, in a<span> </span><a href="https://www.eurekalert.org/news-releases/1004975" target="_blank" rel="noreferrer noopener">press release</a>.</p> <p><strong>RELATED:</strong></p> <ul> <li><a href="https://oxfordglobal.com/resources/targeting-novel-e3-ubiquitin-ligases-carles-galdeano-professor-university-of-barcelona/" target="_blank" rel="noreferrer noopener">Targeting Novel E3 Ubiquitin Ligases: Carles Galdeano, Professor, University of Barcelona</a></li> <li><a href="https://oxfordglobal.com/resources/challenges-in-translational-approaches-and-biomarkers-in-neurodegeneration/" target="_blank" rel="noreferrer noopener">Challenges in Translational Approaches and Biomarkers in Neurodegeneration</a></li> <li><a href="https://oxfordglobal.com/resources/investigating-target-identification-and-validation-modelling-in-neurodegenerative-disease/" target="_blank" rel="noreferrer noopener">Investigating Target Identification and Validation Modelling in Neurodegenerative Disease</a></li> </ul> <p>The preclinical research conducted at UPenn has now garnered enough preclinical data to get a human candidate ready for the clinic. First-in-human clinical trials can begin once the drug has completed IND (investigational new drug)-enabling studies.</p> <p>Allyson Berent, Chief Science Officer at FAST said: “We have been working toward this ‘go or no-go decision’ since 2017. To see the promising results of this robust preclinical data package, based on research that FAST has been so diligently funding for years, is incredibly gratifying.”</p> <h2 id="h-what-is-angelman-syndrome">What is Angelman Syndrome?</h2> <p>Angelman syndrome is a nondegenerative neurogenetic disorder which is caused by a genetic variation on chromosome 15. This difference means that those affected are unable to produce enough of an enzyme called UBE3A (ubiquitin-protein ligase E3A), which assists in the natural degradation of proteins by the body. As a result, those inflicted have problems with motor function, speech, disordered sleeping, and often suffer from seizures.</p> <p>The disorder affects as many as 500,000 people world-wide, and there is currently no cure – only ways to mitigate pain and seizures. Although people with Angelman syndrome have an average life-expectancy, they will need specialist support and care for their whole life due to the challenges that their symptoms pose to independent living.</p> <h2 id="h-aav-gene-therapy">AAV Gene Therapy</h2> <p>Adeno-associated viruses (AAVs) are specialised viruses that can be modified to deliver specific strands of DNA into target cells. On 6 December 2023, the Pennsylvania team revealed that their investigations into the use of AAVs in non-human primates could achieve wanted expression and were “unlikely to cause cancer mutations.”</p> <p>In a press release, Penn Medicine said: “The two new studies, both published today in<span> </span><em><a href="https://www.nature.com/articles/s41587-023-01974-7" target="_blank" rel="noreferrer noopener">Nature Biotechnology</a></em><span> </span>and<span> </span><em><a href="https://doi.org/10.1089/hum.2023.134" target="_blank" rel="noreferrer noopener">Human Gene Therapy</a></em>, amount to the most comprehensive exploration of AAV chromosomal integrations in primates to date and carry important implications for the safety profile and long-term efficacy of AAV-based gene therapies.”</p> <h2 id="h-a-promising-outlook">A Promising Outlook</h2> <p>As well as stoking optimism for people affected by AS, the collaboration has certainly raised hopes for the discovery and development of new therapies of this type.</p> <p>“Our two teams have been both mission and data driven, working closely together to reach this critical inflection point, and FAST and GTP have made an instrumental decision on a promising human clinical candidate to further advance this gene therapy forward quickly and safely for the potential treatment of Angelman syndrome,” commented Wilson.</p> <p>Research into rare diseases can often have trouble receiving both attention and funding, especially rare genetic diseases. However, the collaboration represents a break from this struggle. Berent added:</p> <p>“Too many companies over the past year have been shifting their priorities in the rare disease space, but the priority for FAST will never change. It is our job to ensure that excellent science is always advanced as safely and efficiently as possible, and that we seize every opportunity to potentially benefit those living with Angelman syndrome.”</p> <p><em>Join and network with over 450 industry discovery biology and chemistry leaders at the <a href="https://oxfordglobal.com/discovery-series/" target="_top">renowned Drug Discovery Summit in Basel</a>, where they address critical strategies in target identification, validation<br>and HIT optimisation of small and large molecule drugs.</em></p> <div class="content-body-footer"> </div> </div> <div class="content-sidebar"> <div class="related-articles"> <h3 class="center">Related Resources</h3> <div class="article"> <a href="/discovery-development/resources/cobenfy-a-critical-breakthrough-in-schizophrenia-treatment-in-over-30-years"> <div class="cover-graphic"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Resources/Discovery/FDA%20approves%20Bristol%20Myer%20Squibb%E2%80%99s%20first%20novel%20schizophrenia%20drug%20in%2035%20years.png" class="cover-graphic-image"> </div> </a><div class="content"><a href="/discovery-development/resources/cobenfy-a-critical-breakthrough-in-schizophrenia-treatment-in-over-30-years"> <h4> Cobenfy: A Critical Breakthrough in Schizophrenia Treatment in Over 30 Years </h4> <div class="abstract"> According to a recent press release, the US FDA has approved Cobenfy, a first-in-class muscarinic agonist developed by Bristol Myers Squibb for the treatment of schizophrenia in adults. </div> <div class="read-more"> <a href="/discovery-development/resources/cobenfy-a-critical-breakthrough-in-schizophrenia-treatment-in-over-30-years" class="read-more-link">Read More</a> </div></a> </div> </div> <div class="article"> <a href="/discovery-development/resources/beyond-traditional-therapeutics-how-digital-therapeutics-reshape-neurological-and-neuropsychiatric-treatments"> <div class="cover-graphic"> <img src="https://oxfordglobal.com/hubfs/Copy%20of%20Featured%20Image%20(6).png" class="cover-graphic-image"> </div> </a><div class="content"><a href="/discovery-development/resources/beyond-traditional-therapeutics-how-digital-therapeutics-reshape-neurological-and-neuropsychiatric-treatments"> <h4> Beyond Traditional Therapeutics: How Digital Therapeutics Reshape Neurological and Neuropsychiatric Treatments </h4> <div class="abstract"> Two case studies show how digital therapeutics can potentially treat neurological and neuropsychiatric conditions, areas where conventional therapeutics have encountered obstacles. </div> <div class="read-more"> <a href="/discovery-development/resources/beyond-traditional-therapeutics-how-digital-therapeutics-reshape-neurological-and-neuropsychiatric-treatments" class="read-more-link">Read More</a> </div></a> </div> </div> </div> </div> </div> </div> </div> </div> <div class="newsletter-cta"> <h2 class="heading">Subscribe to our newsletter</h2> <div class="message"> Sign up for our monthly Newsletter to keep up with all things Discovery & Development </div> <div class="button-wrapper"> <a href="#" class="button hs-cta-trigger-button hs-cta-trigger-button-177049184347">Newsletter Sign-Up</a> </div> </div> </main> <div data-global-resource-path="git/og-stage/templates/partials/footer.html"><footer class="website-footer flex flex-column g-20"> <div class="footer-columns"> <div class="footer-column"> <a href="https://oxfordglobal.com" class="site-logo-link"> <img src="https://oxfordglobal.com/wp-content/uploads/2023/03/Oxford-Global-Logo-Stacked-Websize.png" class="site-logo"> </a> </div> <div class="footer-column"> <h3>Brands</h3> <ul> <li> <a href="/precision-medicine/">Precision Medicine</a> </li> <li> <a href="/discovery-development/">Discovery & Development</a> </li> <li> <a href="/nextgen-biomed/">NextGen Biomed</a> </li> </ul> </div> <div class="footer-column"> <h3>Services</h3> <ul> <li> <a href="https://oxfordglobal.com/in-person-services/"> In-Person Services </a> </li> <li> <a href="https://oxfordglobal.com/digital-marketing-services/"> Digital Marketing Services </a> </li> </ul> <h3>Upcoming Events</h3> <ul> <li> <a href="https://oxfordglobal.com/calendar/"> Event Calendar </a> </li> </ul> </div> <div class="footer-column"> <h3>Company</h3> <ul> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/about-us/"> About </a> </li> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/vacancies/"> Vacancies </a> </li> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/csr-policy/"> CSR Policy </a> </li> <li class="elementor-icon-list-item"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});"> Contact </a> </li> </ul> </div> <div class="footer-column"> <p> <a href="tel:+441865248455">+44 1865 248 455</a><br> <a href="mailto:help@oxfordglobal.com">help@oxfordglobal.com</a> </p> <p> <strong>Our UK Address:</strong><br> Godstow Court<br> Minns Business Park<br> Botley<br> Oxford, OX2 0JB </p> <p> <strong>Our US Address:</strong><br> 100 Cambridge Street<br> 14th Floor<br> Boston<br> MA 02114 </p> </div> </div> <div class="footer-bottom grid g-20"> <div class="span8 span12-sm span12-md flex flex-column g-10"> <div class="copyright"> © Oxford Global Marketing Ltd. All rights reserved. </div> <ul class="legal-links flex flex-row g-30 flex-column-sm g-sm-10"> <li><a href="https://oxfordglobal.com/legal/">Terms & Conditions</a></li> <li><a href="https://oxfordglobal.com/legal/privacy-policy/">Privacy Policy</a></li> <li><a href="https://oxfordglobal.com/legal/website-terms/">Cookies</a></li> <li><a href="https://www.wishagency.co.uk/" target="_BLANK">Digital Agency Wish</a></li> </ul> </div> <div class="span4 span12-sm span12-md flex-align-end"> <ul class="social-links flex flex-row flex-justify-end flex-justify-center-sm flex-justify-center-md g-30"> <li><a href="https://www.facebook.com/OGConferences/" target="_BLANK"><i class="fa-brands fa-square-facebook"></i></a></li> <li><a href="https://www.linkedin.com/company/oxford-global/" target="_BLANK"><i class="fa-brands fa-linkedin"></i></a></li> <li><a href="https://twitter.com/OGConferences" target="_BLANK"><i class="fa-brands fa-square-x-twitter"></i></a></li> </ul> </div> </div> </footer></div> </div> <script src="/hs/hsstatic/jquery-libs/static-1.1/jquery/jquery-1.7.1.js"></script> <script>hsjQuery = window['jQuery'];</script>  <script defer src="/hs/hsstatic/content-cwv-embed/static-1.1293/embed.js"></script> <script src="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/182600797781/1731148644227/git/og-stage/js/inlineSVG.min.js"></script> <script src="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/135724366742/1725380923802/git/og-stage/js/main.min.js"></script> <script> function getVideoId(url) { var regExp = /^.*(youtu.be\/|v\/|u\/\w\/|embed\/|watch\?v=|\&v=)([^#\&\?]*).*/; var match = url.match(regExp); if (match && match[2].length == 11) { return match[2]; } else { return false; } } function getVideoEmbed(id) { return '<iframe src="https://www.youtube.com/embed/' + id + '" title="YouTube video player" frameborder="0" allow="accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share" allowfullscreen style="aspect-ratio: 16 / 9; width: 100%;"></iframe>'; } jQuery("div.content-body .wp-block-embed__wrapper:contains('youtu')").each(function() { var yt_url = $(this).html().trim(); var yt_id = getVideoId(yt_url); var yt_embed = getVideoEmbed(yt_id); $(this).html(yt_embed); }); </script>  <script type="text/javascript"> var _hsq = _hsq || []; _hsq.push(["setContentType", "standard-page"]); _hsq.push(["setCanonicalUrl", "https:\/\/oxfordglobal.com\/discovery-development\/resources\/university-of-pennsylvania-and-fast-to-develop-aav-gene-therapy-for-angelman-syndrome"]); _hsq.push(["setPageId", "hubdb-177028096592-7642658-170712574353"]); _hsq.push(["setContentMetadata", { "contentPageId": "hubdb-177028096592-7642658-170712574353", "legacyPageId": "hubdb-177028096592-7642658-170712574353", "contentFolderId": null, "contentGroupId": null, "abTestId": null, "languageVariantId": 177028096592, "languageCode": "en", }]); </script> <script type="text/javascript" id="hs-script-loader" async defer src="/hs/scriptloader/8696823.js"></script>  <script type="text/javascript"> var hsVars = { render_id: "a5b4708e-6246-4363-af6c-e8b906118406", ticks: 1732665700470, page_id: 177028096592, dynamic_page_id: "hubdb-177028096592-7642658-170712574353", content_group_id: 0, portal_id: 8696823, app_hs_base_url: "https://app.hubspot.com", cp_hs_base_url: "https://cp.hubspot.com", language: "en", analytics_page_type: "standard-page", scp_content_type: "", analytics_page_id: "hubdb-177028096592-7642658-170712574353", category_id: 1, folder_id: 0, is_hubspot_user: false } </script> <script defer src="/hs/hsstatic/HubspotToolsMenu/static-1.354/js/index.js"></script> </body></html>

CINXE.COM

University of Pennsylvania and FAST to Develop AAV Gene Therapy for Angelman Syndrome