{"id":956522,"date":"2026-04-30T08:06:49","date_gmt":"2026-04-30T12:06:49","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/"},"modified":"2026-04-30T08:06:49","modified_gmt":"2026-04-30T12:06:49","slug":"solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/","title":{"rendered":"Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy"},"content":{"rendered":"<div class=\"mw_release\">\n<p align=\"center\">\n        <em>&#8211; Solid Biosciences delivers its largest scientific program at the 2026 ASGCT Annual Meeting with 5 oral presentations and 11 posters &#8211;\u00a0<\/em>\n      <\/p>\n<p align=\"justify\">CHARLESTOWN, Mass., April  30, 2026  (GLOBE NEWSWIRE) &#8212; Solid Biosciences Inc. (Nasdaq: SLDB) (the \u201cCompany\u201d or \u201cSolid\u201d), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that multiple abstracts have been accepted for presentation at the upcoming 29<sup>th<\/sup> Annual Meeting of the American Society of Gene &amp; Cell Therapy (ASGCT), taking place May 11-15, 2026, in Boston, MA.<\/p>\n<p align=\"justify\">Solid Biosciences will deliver its largest number of presentations for the Company, reflecting the growing breadth and depth of Solid\u2019s scientific efforts spanning clinical development, next-generation delivery technologies, capsid engineering and scalable manufacturing.<\/p>\n<p align=\"justify\">\u201cWith 16 Solid-led presentations, ASGCT 2026 marks an important meeting for the Company and reflects our growing leadership in next-generation precision genetic medicine,\u201d said Bo Cumbo, President and Chief Executive Officer of Solid Biosciences. \u201cThe number and diversity of presentations we will be sharing underscore how far our organization has progressed in three short years, in addition to the scientific rigor we\u2019re applying across discovery, development, manufacturing and clinical translation. From advanced platform and delivery technology engineering to updates from our clinical programs, this body of work reflects both the ambition of our team and the increasing maturity of our science.\u201d<\/p>\n<p align=\"justify\">\n        <strong>Oral Presentations<\/strong>\n      <\/p>\n<ol>\n<li>\n          <strong>Title:<\/strong> Update on the INSPIRE DUCHENNE Phase 1\/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy<br \/><strong>Session:<\/strong> In vivo clinical trials in eye and muscle disorders<br \/><strong>Presentation ID:<\/strong> 502<br \/><strong>Date\/Time:<\/strong> May 15, 3:30pm ET<br \/><strong>Presenter:<\/strong> Kevin Flanigan, MD, Advisor Consultant &amp; Researcher, Nationwide Children\u2019s Hospital and Principal Investigator in the Phase 1\/2 INSPIRE DUCHENNE clinical trial<\/li>\n<li>\n          <strong>Title:<\/strong> Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich\u2019s Ataxia<br \/><strong>Session:<\/strong> Neurologic diseases V<br \/><strong>Presentation ID:<\/strong> 353<br \/><strong>Date\/Time:<\/strong> May 14, 10:30am ET<br \/><strong>Presenter:<\/strong> Brandon Chan, PhD, Discovery &amp; Translational Development, Scientific Lead<\/li>\n<li>\n          <strong>Title:<\/strong> Development and Implementation of a High Yield and Scalable Manufacturing Process for Adeno-Associated Virus (AAV) Production<br \/><strong>Session:<\/strong> Scale-up, scale-out, formulation, supply chains, quality, and logistics I<br \/><strong>Presentation ID:<\/strong> 246<br \/><strong>Date\/Time:<\/strong> May 13, 4:00pm ET<br \/><strong>Presenter:<\/strong> Ben Wright, Vice President, Head of Process Development<\/li>\n<li>\n          <strong>Title:<\/strong> Boosting rAAV Yield in HEK293 Cells Through Multi-Mechanism Strategies<br \/><strong>Session:<\/strong> AAV transfection improvements: Scaling and titers<br \/><strong>Presentation ID:<\/strong> 320<br \/><strong>Date\/Time:<\/strong> May 14, 10:30am ET<br \/><strong>Presenter:<\/strong> Xiaofei E, PhD, Director, Molecular Therapeutics<\/li>\n<li>\n          <strong>Title:<\/strong> Utilizing Machine Learning and Mechanistic Understanding to Appreciate the Impact of pH, Dissolved Oxygen, and pCO\u2082 on Upstream AAV Yield and Product Quality<br \/><strong>Session: <\/strong>AAV critical quality attributes<br \/><strong>Presentation ID:\u00a0<\/strong>263<br \/><strong>Date\/Time: <\/strong>May 14, 8:00am ET<br \/><strong>Presenter:<\/strong> Neil Templeton, PhD, Director, Upstream Process Development<\/li>\n<\/ol>\n<p align=\"justify\">\n        <strong>Poster Presentations<\/strong>\n      <\/p>\n<ol>\n<li>\n          <strong>Title:\u00a0<\/strong>Novel Pharmacokinetic Effects of POLARIS-101\u2122\u00a0(AAV-SLB101), Solid Biosciences\u2019 Rationally Designed, Next-Generation Capsid<br \/><strong>Presentation ID:<\/strong> 3385<br \/><strong>Presenter:<\/strong> Patrick Gonzalez, PhD, Vice President, Head of Clinical Science<\/li>\n<li>\n          <strong>Title:<\/strong> SGT-003 Gene Therapy Restores DAPC Stability and Improves Muscle Integrity in Duchenne Muscular Dystrophy<br \/><strong>Presentation ID:<\/strong> 2365<br \/><strong>Presenter:<\/strong> Jamie Marshall, PhD, Director and Scientific Lead, Discovery &amp; Translational Development<\/li>\n<li>\n          <strong>Title:<\/strong> A Novel AAV Gene Therapy Strategy to Correct Calcium Dysregulation in Catecholaminergic Polymorphic Ventricular Tachycardia <br \/><strong>Presentation ID:<\/strong> 1429<br \/><strong>Presenter:<\/strong> Meghan Soustek-Kramer, PhD, Principal Scientist and Scientific Lead, Discovery &amp; Translational Development<\/li>\n<li>\n          <strong>Title:<\/strong> Systemic Delivery of Human TNNT2 Gene Therapy Using the Novel Capsid POLARIS-101\u2122\u00a0(AAV-SLB101) Improves Cardiac Function in the TNNT2 R141W Knock-in Mouse Model of Dilated Cardiomyopathy<br \/><strong>Presentation ID:<\/strong> 3430<br \/><strong>Presenter:<\/strong> Jamie Marshall, PhD, Director and Scientific Lead, Discovery &amp; Translational Development<\/li>\n<li>\n          <strong>Title:<\/strong> Systemic Administration of a Rationally Engineered Vector (POLARIS-101\u2122\u00a0[AAV-SLB101]) Leads to Successful Transduction of Non-Human Primate Tissues in the Presence of Neutralizing Antibodies <br \/><strong>Presentation ID:<\/strong> 3397<br \/><strong>Presenter:<\/strong> Kruti Patel, PhD, Associate Director and Scientific Lead, Discovery &amp; Translational Development<\/li>\n<li>\n          <strong>Title:<\/strong> Lower POLARIS-101<sup>TM<\/sup> (AAV-SLB101) Cross-Reactive Antibodies in Elevidys-Treated Patients<br \/><strong>Presentation ID:<\/strong> 3382<br \/><strong>Presenter:<\/strong> Marla Bazile, Principal Associate Scientist, R&amp;D<\/li>\n<li>\n          <strong>Title:<\/strong> Small Molecule Induced G2\/M Arrest Boosts Recombinant AAV Production and Preserves High Quality <br \/><strong>Presentation ID:\u00a0<\/strong>3146<br \/><strong>Presenter:<\/strong> Xiaofei E, PhD, Director, Molecular Therapeutics<\/li>\n<li>\n          <strong>Title:<\/strong> Impact of Genome Size and Sequence Composition on AAV Vector Genome Integrity<br \/><strong>Presentation ID:<\/strong> 2010<br \/><strong>Presenter:<\/strong> Jie Tan, PhD, Director, Predictive Sciences<\/li>\n<li>\n          <strong>Title:<\/strong> Connecting Changes in AAV Capsid Protein Charge Species to In Vitro Protein Expression<br \/><strong>Presentation ID: <\/strong>2216<br \/><strong>Presenter:<\/strong> Jonathan Hill, PhD, Principal Scientist<\/li>\n<li>\n          <strong>Title:<\/strong> Impact of Full vs. Empty AAV Capsids on Vector Potency<br \/><strong>Presentation ID:<\/strong> 2221<br \/><strong>Presenter:<\/strong> Cristiano Vieira, PhD, Senior Scientist, Analytical<\/li>\n<li>\n          <strong>Title:<\/strong> Identifying Sources of Library Preparation Artifacts in AAV Vectors with SMRT Sequencing<br \/><strong>Presentation ID:<\/strong> 1219<br \/><strong>Presenter:<\/strong> Sarah Tong, Senior Associate Scientist, Analytical Development<\/li>\n<\/ol>\n<p align=\"justify\">Additionally, Armatus Bio will present a poster highlighting POLARIS-101\u2122\u2019s utility in Facioscapulohumeral muscular dystrophy (FSHD) animal models:<\/p>\n<ul>\n<li>\n          <strong>Title<\/strong><br \/>\n          <em>: <\/em>FSHD Gene Therapy Using Systemic Delivery of Therapeutic miRNAs and the Engineered AAV-SLB101 Capsid Corrects FSHD-associated Phenotypes at Lower Doses Than First Generation Vectors<\/li>\n<li>\n          <strong>Presentation ID<\/strong><br \/>\n          <em>: <\/em>1500<\/li>\n<li>\n          <strong>Presenter: <\/strong>Lindsay Wallace, PhD, Center for Gene Therapy, The Research Institute at Nationwide Children\u2019s Hospital<\/li>\n<\/ul>\n<p align=\"justify\">\n        <strong>About Solid Biosciences<\/strong><br \/>\n        <br \/>Solid Biosciences\u00a0is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich\u2019s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid\u2019s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=BnaDFSOuiYz4p-OdnKBfVI9GXnFIAwijhRomgg0nMn2_mMJ9pM0XV6OxaIvUvbWjAJ3-mJi0bNT8kBQ8SyDrK-iETztz0qS7VhrtbgeO0bR5XyFJ2GzxeEYBaYj8Kwj3AXQBWl0OGvRDyZVRwj47hDyv1xMi6qOCOE4eCl45dQ4ZWcte-KKv2SkI3k1EjRX2dB8VZa356xWx3tFXG6dWNZSmKXn3qwVB18d-ONKv-91BuMzshPT3R7SoAVY_MhaQW--6wRHdgo8KRkFqTetJTA==\" rel=\"nofollow\" target=\"_blank\">www.solidbio.com<\/a>.<\/p>\n<p align=\"justify\">\n        <strong>Cautionary Note Regarding Forward-Looking Statements<\/strong><br \/>\n        <br \/>This press release contains \u201cforward-looking statements\u201d within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company\u2019s goals; anticipated benefits of and strategies and expectations for the company\u2019s SGT-003, SGT-212 and SGT-501 programs; expectations for planned enrollment of its SGT-003, SGT-212 and SGT-501 programs; planned regulatory interactions and the potential approval pathways for SGT-003; and other statements containing the words \u201canticipate,\u201d \u201cbelieve,\u201d \u201ccontinue,\u201d \u201ccould,\u201d \u201cestimate,\u201d \u201cexpect,\u201d \u201cintend,\u201d \u201cmay,\u201d \u201cplan,\u201d \u201cpotential,\u201d \u201cpredict,\u201d \u201cproject,\u201d \u201cshould,\u201d \u201ctarget,\u201d \u201cwould,\u201d \u201cworking\u201d and similar expressions. Any forward-looking statements are based on management\u2019s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company\u2019s ability to advance SGT-003, SGT-212, SGT-501, SGT-601 and other preclinical programs, capsid libraries and other enabling technologies on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company\u2019s product candidates; manufacture sufficient quantities of our drug product in a timely manner and maintain adequate supply to support our clinical development and potential commercialization; obtain, maintain or protect intellectual property rights related to its product candidates; replicate preliminary or interim data from clinicals trials in the final data of such trials; compete successfully with other companies that are seeking to develop Duchenne, FA, CPVT and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-212, SGT-501, SGT-601 and other candidates; achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company\u2019s actual results to differ from those contained in the forward-looking statements, see the \u201cRisk Factors\u201d section, as well as discussions of potential risks, uncertainties and other important factors, in the company\u2019s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company\u2019s views as of the date hereof and should not be relied upon as representing the company\u2019s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company&#8217;s views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.<\/p>\n<p align=\"justify\">\n        <strong>Solid Biosciences Investor Contact:<\/strong><br \/>\n        <br \/>Nicole Anderson<br \/>Senior Director, Investor Relations and Corporate Communications<br \/>Solid Biosciences Inc.<br \/><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=XhOlCblz31xKruadz0naXZR67mduvp-2xk36lVfOofdfi8zdM7raj0rbYhueWiIUfjBYlWuq3IubGSGF00Daa38AHzfNDzv7bmyA15nNnC-VxAlMViuc3HTeh_Jy5BZziQQOX4q1YK5XiP6QdgYLI1-OUguufKWtyPQyeIGOz-c6ebxovGxttL7xlXdkssaEsvuNk8Ts5ucPSsvT_2eNGllL_FGJg-A5Fi9oW5E5lO5X9JChPrH7vw3wXm0T9AB-kvlpiC_KLuOWiS6i1xOCeg==\" rel=\"nofollow\" target=\"_blank\"><u>investors@solidbio.com<\/u><\/a><\/p>\n<p>      <img decoding=\"async\" alt=\"\" class=\"__GNW8366DE3E__IMG\" src=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=OTcxMDA4OSM3NTc0MzE3IzIwOTExOTc=\" \/><br \/>\n      <br \/>\n      <img decoding=\"async\" alt=\"\" src=\"https:\/\/ml.globenewswire.com\/media\/MmQ5YzBkODItNzYwNS00YjUxLWE1M2MtZjA5MWJkZjBhYmNkLTExMDI3NjgtMjAyNi0wNC0zMC1lbg==\/tiny\/Solid-Biosciences-Inc-.png\" \/>\n    <\/div>\n<div class=\"mw_contactinfo\"><\/div>\n","protected":false},"excerpt":{"rendered":"<p>&#8211; Solid Biosciences delivers its largest scientific program at the 2026 ASGCT Annual Meeting with 5 oral presentations and 11 posters &#8211;\u00a0 CHARLESTOWN, Mass., April 30, 2026 (GLOBE NEWSWIRE) &#8212; Solid Biosciences Inc. (Nasdaq: SLDB) (the \u201cCompany\u201d or \u201cSolid\u201d), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that multiple abstracts have been accepted for presentation at the upcoming 29th Annual Meeting of the American Society of Gene &amp; Cell Therapy (ASGCT), taking place May 11-15, 2026, in Boston, MA. Solid Biosciences will deliver its largest number of presentations for the Company, reflecting the growing breadth and depth of Solid\u2019s scientific efforts spanning clinical development, next-generation delivery technologies, capsid engineering and scalable manufacturing. \u201cWith &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/www.marketnewsdesk.com\/index.php\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy&#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-956522","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy - 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\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"&#8211; Solid Biosciences delivers its largest scientific program at the 2026 ASGCT Annual Meeting with 5 oral presentations and 11 posters &#8211;\u00a0 CHARLESTOWN, Mass., April 30, 2026 (GLOBE NEWSWIRE) &#8212; Solid Biosciences Inc. (Nasdaq: SLDB) (the \u201cCompany\u201d or \u201cSolid\u201d), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that multiple abstracts have been accepted for presentation at the upcoming 29th Annual Meeting of the American Society of Gene &amp; Cell Therapy (ASGCT), taking place May 11-15, 2026, in Boston, MA. Solid Biosciences will deliver its largest number of presentations for the Company, reflecting the growing breadth and depth of Solid\u2019s scientific efforts spanning clinical development, next-generation delivery technologies, capsid engineering and scalable manufacturing. \u201cWith &hellip; Continue reading &quot;Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy&quot;\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.marketnewsdesk.com\/index.php\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/\" \/>\n<meta property=\"og:site_name\" content=\"Market Newsdesk\" \/>\n<meta property=\"article:published_time\" content=\"2026-04-30T12:06:49+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=OTcxMDA4OSM3NTc0MzE3IzIwOTExOTc=\" \/>\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=\"7 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\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/\"},\"author\":{\"name\":\"Newsdesk\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/#\\\/schema\\\/person\\\/482f27a394d4fda80ecb5499e519d979\"},\"headline\":\"Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy\",\"datePublished\":\"2026-04-30T12:06:49+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/\"},\"wordCount\":1445,\"image\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.globenewswire.com\\\/newsroom\\\/ti?nf=OTcxMDA4OSM3NTc0MzE3IzIwOTExOTc=\",\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/\",\"url\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/solid-biosciences-to-showcase-a-large-presence-at-the-29th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\\\/\",\"name\":\"Solid Biosciences to Showcase a Large Presence at the 29th Annual Meeting of the American Society of Gene and Cell Therapy - 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