{"id":491030,"date":"2021-05-11T07:33:37","date_gmt":"2021-05-11T11:33:37","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/"},"modified":"2021-05-11T07:33:37","modified_gmt":"2021-05-11T11:33:37","slug":"voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/","title":{"rendered":"Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates"},"content":{"rendered":"<div class=\"mw_release\">\n<ul>\n<li style=\"text-align:left\">\n          <em>Data to be presented at the 24<\/em><br \/>\n          <sup><br \/>\n            <em>th<\/em><br \/>\n          <\/sup><br \/>\n          <em> Annual Meeting of the American Society of Gene and Cell Therapy show up to 1000-fold higher transgene expression in the brain compared with conventional AAV gene therapy following IV administration<\/em>\n        <\/li>\n<\/ul>\n<p>CAMBRIDGE, Mass., May  11, 2021  (GLOBE NEWSWIRE) &#8212; Voyager Therapeutics, Inc.\u00a0(NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for serious neurological diseases, today will present new preclinical data demonstrating high transduction efficiency of the company\u2019s novel adeno-associated virus (AAV) capsids in the central nervous system (CNS) after intravenous dosing in non-human primates. Data will be presented at the 24<sup>th<\/sup> Annual Meeting of the American Society of Gene\u00a0and\u00a0Cell Therapy\u00a0(ASGCT) taking place virtually May 11-14, 2021.<\/p>\n<p>\u201cEfficient targeting of the CNS, including the ability to penetrate the blood brain barrier and transduce target cells, have represented a significant challenge in gene therapy hindering the field\u2019s ability to effectively address many serious neurological diseases,\u201d Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of Research &amp; Development at Voyager. \u201cWe\u2019ve now been able to show that our novel capsid variants not only cross the blood brain barrier but achieve widespread transduction of multiple brain regions including the cortex, thalamus, striatum, cerebellum, brainstem and spinal cord. We believe these capsids can provide a new way forward to treat a broad range of CNS indications, potentially at significantly lower doses than currently available AAV serotypes.\u201d<\/p>\n<p>Data will be shared in an oral presentation titled, \u201cRNA-driven Evolution of AAV Capsid Libraries Identifies Variants\u00a0with High Transduction Efficiency in Non-Human Primate Central\u00a0Nervous System,\u200b\u201d (Abstract #51) by Mathieu Nonnenmacher, Ph.D., Director and Head of Capsid Discovery, showing that several capsid variants derived from the company\u2019s directed evolution RNA-based screening platform, TRACER<sup>TM<\/sup> (Tropism Redirection of AAV by Cell-type-specific Expression of RNA), demonstrated significantly enhanced activity relative to AAV9, the most commonly used vector for CNS gene therapy.<\/p>\n<ul type=\"disc\">\n<li style=\"margin-bottom:8pt\">A subset of capsids showed a 10-fold or higher improvement in transduction of the brain and spinal cord, compared to AAV9.<\/li>\n<li style=\"margin-bottom:8pt\">The most efficient capsid identified in the work to be presented, TRACER 9P801, displayed more than 1,000-fold higher transgene expression in the brain and 100-fold higher transgene expression in the spinal cord.<\/li>\n<li style=\"margin-bottom:8pt\">The overall tolerability of 9P801 was favorable and no toxicity was observed in the liver, spinal cord or dorsal root ganglia (DRG).<\/li>\n<li style=\"margin-bottom:8pt\">Immunohistochemical analysis indicated that 9P801 displayed predominant neuronal tropism and achieved widespread transduction of multiple brain regions including the cortex, thalamus, putamen and brainstem.<\/li>\n<li style=\"margin-bottom:8pt\">The TRACER platform generates large data sets on engineered capsid performance in a relevant primate species suitable for in silico approaches to optimizing capsid selection for specific cellular targeting and tropism.<\/li>\n<\/ul>\n<p>Voyager\u2019s TRACER system is a broadly-applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV capsids with cell-specific transduction properties in wild-type animals. TRACER candidates were tested individually by low dose intravenous injection and their tropism for the CNS analyzed by measuring transgene RNA expression, viral DNA biodistribution and immunohistochemistry.<\/p>\n<p>Further information about the Company\u2019s TRACER platform and pipeline, including novel capsid-enabled new programs, will be shared at an upcoming investor and analyst event in July 2021.<\/p>\n<p>\n        <strong>About\u00a0Voyager Therapeutics<\/strong>\n      <\/p>\n<p>Voyager Therapeutics\u00a0is a clinical-stage gene therapy company focused on developing life-changing treatments for serious neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. For more information on\u00a0Voyager Therapeutics, please visit the company\u2019s website at\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=SgVvOb3qd5hDqmPZHAfsMdo22cS_HDqrK9U2n1_gZznfZBkKj_CQbDiRcctTXIN1BoFLEVqCM7JSRZgi6gigZ9ai-wUi7SbpwxEzP0qWq_zy7yjgTicjdGlyTGaOKF1J\" rel=\"nofollow noopener\" target=\"_blank\">www.voyagertherapeutics.com<\/a>\u00a0or follow\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=Yp55XItPPl1ZNHNUQgs78UTEMFs811Tdtp78No5fwpicmc4CMpFI3Le07GCGzM1JQrvtu2pRx7El4ueld32P1A==\" rel=\"nofollow noopener\" target=\"_blank\">@VoyagerTx<\/a>\u00a0on Twitter and\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=5Ewm9C2WXFW6YPmTXbbLHXJ1QVwpF9OQF6ikS8DgGbM-bGjw_tk3GY71vYlfK-8y9gz3iI9VRV66uPWK44Dktb-TTDkMYyuRxJ5Drj44oNFM7JXHwtGTxUfinZ_e5TtZ\" rel=\"nofollow noopener\" target=\"_blank\">LinkedIn<\/a>.<\/p>\n<p>\n        <em>Voyager Therapeutics<sup>\u00ae<\/sup> is a registered trademark, and TRACER\u2122 is a trademark, of\u00a0Voyager Therapeutics, Inc.\u00a0<\/em>\n      <\/p>\n<p>\n        <strong>Forward-Looking Statements<\/strong>\n      <\/p>\n<p>This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as \u201cmay,\u201d \u201cmight,\u201d \u201cwill,\u201d \u201cwould,\u201d \u201cshould,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201canticipate,\u201d \u201cbelieve,\u201d \u201cestimate,\u201d \u201cundoubtedly,\u201d \u201cproject,\u201d \u201cintend,\u201d \u201cfuture,\u201d \u201cpotential,\u201d or \u201ccontinue,\u201d and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress, activities, goals and reporting of results of its research and development programs; Voyager\u2019s ability to present preclinical data on its early pipeline progress and novel capsid discovery efforts in non-human primates at scientific meetings and other presentations; Voyager\u2019s ability to continue to develop its novel AAV capsids; the ability of Voyager\u2019s novel capsids to treat CNS diseases and to treat with lower doses than currently available AAV serotypes, in each instance are forward looking.<\/p>\n<p>All forward-looking statements are based on estimates and assumptions by Voyager\u2019s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the severity and length of the COVID-19 health crisis; the initiation and conduct of preclinical studies involving our novel capsid program; the continued development of the Voyager gene therapy platform and Voyager\u2019s TRACER system; the applicability of novel capsids identified by the TRACER system to a broad spectrum of CNS and non-CNS disease indications; Voyager\u2019s scientific approach and general development progress; the ability to attract and retain highly\u00a0 skilled and experienced scientists and technicians; the ability to create and protect intellectual property related to Voyager\u2019s development of novel capsids; and the availability of resources necessary to conduct the novel capsid development program, including access to sufficient number and types of primates suitable for participation in ongoing and planned preclinical research activities.<\/p>\n<p>These statements are also subject to a number of material risks and uncertainties that are described in Voyager\u2019s Annual Report on Form 10-K for the year ended December 31, 2020 filed with the\u00a0Securities and Exchange Commission, as updated by its subsequent filings with the\u00a0Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.<\/p>\n<p>\n        <strong>Investor Inquiries:<\/strong>\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0<br \/><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=UXfFiM8k-SFkKTNwTFRZh6gGsZQ_UkNRKiFy9wRi-zqllovQyw9PpQWwSq2WNaj4ZZPtxNwmVUlgufqanaAtR0NuOZwnK7QwzgqUF9wpCjZthtJqXHsTOrXU6H3xCbor\" rel=\"nofollow noopener\" target=\"_blank\">investors@voyagertherapeutics.com<\/a><\/p>\n<p>\n        <strong>Media Inquiries:<\/strong>\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0<br \/>Lissette Steele<br \/>Verge Scientific Communications<br \/>202.930.4762 x 409<br \/><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=RhMwkeQB6uHL_bSIv9pCxqa5P6VDDh5jZ6_J-rTWupF_Gp3Kj3xcdxhyhaUYg7OpmGF-_13uriozUa7V6-Mp9ipa2_npMSHLiZZ8q8kzzD005FJAiHooKVZqLtCRKmym\" rel=\"nofollow noopener\" target=\"_blank\">lsteele@vergescientific.com<\/a><\/p>\n<p>      <img loading=\"lazy\" decoding=\"async\" class=\"__GNW8366DE3E__IMG\" src=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=ODIzMzY3MCM0MTc2NDgwIzIwMTg4NDA=\" width=\"1\" height=\"1\" \/><br \/>\n      <br \/>\n      <img loading=\"lazy\" decoding=\"async\" class=\"__GNW8366DE3E__IMG\" src=\"https:\/\/ml.globenewswire.com\/release\/track\/4a7ef65b-d80e-487c-ba66-c4d5f2b17729\" width=\"1\" height=\"1\" \/>\n    <\/div>\n<div class=\"mw_contactinfo\"><\/div>\n","protected":false},"excerpt":{"rendered":"<p>Data to be presented at the 24 th Annual Meeting of the American Society of Gene and Cell Therapy show up to 1000-fold higher transgene expression in the brain compared with conventional AAV gene therapy following IV administration CAMBRIDGE, Mass., May 11, 2021 (GLOBE NEWSWIRE) &#8212; Voyager Therapeutics, Inc.\u00a0(NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for serious neurological diseases, today will present new preclinical data demonstrating high transduction efficiency of the company\u2019s novel adeno-associated virus (AAV) capsids in the central nervous system (CNS) after intravenous dosing in non-human primates. Data will be presented at the 24th Annual Meeting of the American Society of Gene\u00a0and\u00a0Cell Therapy\u00a0(ASGCT) taking place virtually May 11-14, 2021. \u201cEfficient targeting of the &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/www.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates&#8221;<\/span><\/a><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-491030","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates - Market Newsdesk<\/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.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"Data to be presented at the 24 th Annual Meeting of the American Society of Gene and Cell Therapy show up to 1000-fold higher transgene expression in the brain compared with conventional AAV gene therapy following IV administration CAMBRIDGE, Mass., May 11, 2021 (GLOBE NEWSWIRE) &#8212; Voyager Therapeutics, Inc.\u00a0(NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for serious neurological diseases, today will present new preclinical data demonstrating high transduction efficiency of the company\u2019s novel adeno-associated virus (AAV) capsids in the central nervous system (CNS) after intravenous dosing in non-human primates. Data will be presented at the 24th Annual Meeting of the American Society of Gene\u00a0and\u00a0Cell Therapy\u00a0(ASGCT) taking place virtually May 11-14, 2021. \u201cEfficient targeting of the &hellip; Continue reading &quot;Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates&quot;\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/\" \/>\n<meta property=\"og:site_name\" content=\"Market Newsdesk\" \/>\n<meta property=\"article:published_time\" content=\"2021-05-11T11:33:37+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=ODIzMzY3MCM0MTc2NDgwIzIwMTg4NDA=\" \/>\n<meta name=\"author\" content=\"Newsdesk\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Newsdesk\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"5 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/\"},\"author\":{\"name\":\"Newsdesk\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/#\\\/schema\\\/person\\\/482f27a394d4fda80ecb5499e519d979\"},\"headline\":\"Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates\",\"datePublished\":\"2021-05-11T11:33:37+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/\"},\"wordCount\":1084,\"image\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.globenewswire.com\\\/newsroom\\\/ti?nf=ODIzMzY3MCM0MTc2NDgwIzIwMTg4NDA=\",\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/\",\"url\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\\\/\",\"name\":\"Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates - 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Market Newsdesk","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/www.marketnewsdesk.com\/index.php\/voyager-therapeutics-novel-capsids-demonstrate-robust-delivery-across-the-blood-brain-barrier-widespread-cns-transduction-in-non-human-primates\/","og_locale":"en_US","og_type":"article","og_title":"Voyager Therapeutics\u2019 Novel Capsids Demonstrate Robust Delivery Across the Blood Brain Barrier, Widespread CNS Transduction in Non-Human Primates - Market Newsdesk","og_description":"Data to be presented at the 24 th Annual Meeting of the American Society of Gene and Cell Therapy show up to 1000-fold higher transgene expression in the brain compared with conventional AAV gene therapy following IV administration CAMBRIDGE, Mass., May 11, 2021 (GLOBE NEWSWIRE) &#8212; Voyager Therapeutics, Inc.\u00a0(NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for serious neurological diseases, today will present new preclinical data demonstrating high transduction efficiency of the company\u2019s novel adeno-associated virus (AAV) capsids in the central nervous system (CNS) after intravenous dosing in non-human primates. 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