{"id":5484,"date":"2026-04-28T11:00:00","date_gmt":"2026-04-28T11:00:00","guid":{"rendered":"https:\/\/airna.com\/news\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\/"},"modified":"2026-08-19T20:40:48","modified_gmt":"2026-08-19T20:40:48","slug":"airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting","status":"publish","type":"post","link":"https:\/\/airna.com\/de\/news\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\/","title":{"rendered":"AIRNA stellt auf der ASGCT-Jahrestagung neue pr\u00e4klinische Daten zur therapeutischen RNA-Editing-Pipeline vor"},"content":{"rendered":"\n<ul class=\"wp-block-list\">\n<li><em>In-vivo-Daten unterst\u00fctzen ein potenzielles Best-in-Class-Profil f\u00fcr AIR-001, das derzeit in einer klinischen Phase-I-Studie zu Alpha-1-Antitrypsin-Defizienz (AATD) untersucht wird<\/em><\/li>\n\n\n\n<li><em>Daten aus dem kardiometabolischen Portfolio zeigen das Potenzial des AIRNA-Ansatzes zur Einf\u00fchrung vorteilhafter Varianten<\/em><\/li>\n<\/ul>\n\n<p class=\"wp-block-paragraph\"><strong>CAMBRIDGE, Mass., 28. April 2026 <\/strong>\u2013 <a href=\"https:\/\/airna.com\/de\/\">AIRNA<\/a>, ein Biotech-Unternehmen, das Pionierarbeit bei RNA-Editing-Therapeutika leistet, die die Lebenssituation von Patienten sowohl mit seltenen als auch h\u00e4ufigen Erkrankungen verbessern sollen, wird auf der Jahrestagung 2026 der American Society of Gene and Cell Therapy (ASGCT) neue pr\u00e4klinische Daten aus der Pipeline von RNA-Editing-Therapeutika des Unternehmens, einschlie\u00dflich des Leitkandidaten AIR-001 und des kardiometabolischen Portfolios des Unternehmens, pr\u00e4sentieren. <\/p>\n\n<p class=\"wp-block-paragraph\">In einem Vortrag wird AIRNA die ersten in-vivo-Daten zu AIR-001, dem f\u00fchrenden therapeutischen RNA-Editing-Kandidaten von AIRNA f\u00fcr Alpha-1-Antitrypsin-Defizienz (AATD), pr\u00e4sentieren. AIR-001 ist ein subkutan verabreichtes GalNAc-Oligonukleotid, das daf\u00fcr konzipiert ist, die h\u00e4ufigste krankheitsverursachende Mutation des AATD (PiZ) auf RNA-Ebene zu korrigieren, indem es endogene ADAR-Enzyme rekrutiert, um die SERPINA1-mRNA pr\u00e4zise zu editieren. AIR-001 wird derzeit in der klinischen Phase-I-Studie RepAIR1 (<a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07431112\">NCT07431112<\/a>)getestet.  <\/p>\n\n<p class=\"wp-block-paragraph\">Dar\u00fcber hinaus wird das Unternehmen in einer Posterpr\u00e4sentation bei ASGCT pr\u00e4klinische Machbarkeitsstudien vorstellen, in denen RNA-Editierung genutzt wird, um nat\u00fcrlich vorkommende Schutzvarianten in die Transkripte von zwei Genen einzuf\u00fchren, die an kardiovaskul\u00e4ren Erkrankungen beteiligt sind: dem Low-Density-Lipoprotein-Rezeptor (LDLR) und Apolipoprotein B (APOB). Diese Erkenntnisse unterstreichen die Breite der RNA-Editing-Plattform von AIRNA, die das Potenzial hat, auf RNA-Ebene pr\u00e4zise positive Ver\u00e4nderungen einzuleiten. <\/p>\n\n<p class=\"wp-block-paragraph\">N\u00e4heres \u00fcber die Pr\u00e4sentationen:<\/p>\n\n<ul class=\"wp-block-list\">\n<li><strong>Vortragstitel<\/strong>: \u201eAIR-001: A highly potent and precise GalNAc-conjugated RNA base editing treatment for alpha-1 antitrypsin deficiency\u201c<\/li>\n\n\n\n<li><strong>Abstract-Nummer<\/strong>: 77<\/li>\n\n\n\n<li><strong>Sitzung<\/strong>: Translational Gene Therapy and Editing in Storage Disorders<\/li>\n\n\n\n<li><strong>Datum\/Uhrzeit<\/strong>: Dienstag, 12. Mai, 11:15\u201311:30 Uhr ET<\/li>\n\n\n\n<li><strong>Ort<\/strong>: MCEC Raum 258ABC<\/li>\n<\/ul>\n\n<ul class=\"wp-block-list\">\n<li><strong>Titel der Posterpr\u00e4sentation<\/strong>: \u201eRNA Editing: A Modality for Precise Introduction of Gain-of-Function Variants Identified by Human Genetics\u201c<\/li>\n\n\n\n<li><strong>Abstract-Nummer<\/strong>: 1426<\/li>\n\n\n\n<li><strong>Sitzung<\/strong>: Posterempfang<\/li>\n\n\n\n<li><strong>Datum\/Uhrzeit<\/strong>: Dienstag, 12. Mai, 17:00\u201318:30 Uhr<\/li>\n\n\n\n<li><strong>Ort<\/strong>: MCEC-Ausstellungs- und Posterhalle B2-C, Ausstellungsebene<\/li>\n<\/ul>\n\n<p class=\"wp-block-paragraph\"><strong>\u00dcber AIR-001<\/strong><\/p>\n\n<p class=\"wp-block-paragraph\">AIR-001 ist der f\u00fchrende therapeutische RNA-Editing-Kandidat von AIRNA f\u00fcr die Alpha-1-Antitrypsin-Defizienz (AATD), einer genetisch bedingten Erkrankung, die meist durch die PiZ-Mutation im SERPINA1-Gen verursacht wird. AIR-001 soll die zugrundeliegende Ursache der Erkrankung beheben und die Produktion des funktionellen Alpha-1-Antitrypsin-Proteins wiederherstellen, mit dem Ziel, sowohl die Lungen- als auch die Lebermanifestationen des AATM zu behandeln. AIR-001 wird durch subkutane Injektion verabreicht und ist f\u00fcr eine unkomplizierte, wiederholte Verabreichung geeignet. AIR-001 hat von der US-amerikanischen Arzneimittelbeh\u00f6rde FDA den Status eines Arzneimittels f\u00fcr seltene Krankheiten zur Behandlung der Alpha-1-Antitrypsin-Defizienz erhalten und wird derzeit in der klinischen Phase-I-Studie RepAIR1 (<a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07431112\">NCT07431112<\/a>) getestet.   <\/p>\n\n<p class=\"wp-block-paragraph\"><strong>\u00dcber AIRNA<\/strong><\/p>\n\n<p class=\"wp-block-paragraph\">AIRNA ist ein Unternehmen f\u00fcr Arzneimittel in der Phase der klinischen Pr\u00fcfung, das RNA-Editierungsmedikamente entwickelt, die darauf abzielen, krankheitsverursachende genetische Mutationen zu reparieren und vorteilhafte Mutationen herbeizuf\u00fchren, die die menschliche Gesundheit f\u00f6rdern. Die firmeneigene Plattform basiert auf Forschungsarbeiten der Pioniere der RNA-Editierung und Mitbegr\u00fcnder Thorsten Stafforst (Universit\u00e4t T\u00fcbingen) und Jin Billy Li (Stanford University). AIRNA arbeitet an einer Reihe von RNA-Editierungstherapeutika, allen voran ein Produktkandidat f\u00fcr den Alpha-1-Antitrypsin-Mangel (AATM). AIRNA hat seinen Hauptsitz in Cambridge, Massachusetts, und unterh\u00e4lt eine Forschungsniederlassung in T\u00fcbingen, Deutschland. Weitere Informationen erhalten Sie unter <a href=\"https:\/\/airna.com\/de\/\">https:\/\/airna.com\/<\/a>.    <\/p>\n\n<p class=\"wp-block-paragraph\"><strong>Medien:<\/strong><\/p>\n\n<p class=\"wp-block-paragraph\">Michael Galfetti<br\/>Ten Bridge Communications<br\/><a href=\"mailto:tbcAIRNA@tenbridgecommunications.com\" target=\"_blank\" rel=\"noreferrer noopener\">tbcAIRNA@tenbridgecommunications.com<\/a><\/p>\n\n<p class=\"wp-block-paragraph\"><\/p>\n","protected":false},"excerpt":{"rendered":"<p>CAMBRIDGE, Mass., 28. April 2026 \u2013 AIRNA, ein Biotech-Unternehmen, das Pionierarbeit bei RNA-Editing-Therapeutika leistet, die die Lebenssituation von Patienten sowohl mit seltenen als auch h\u00e4ufigen Erkrankungen verbessern sollen, wird auf der Jahrestagung 2026 der American Society of Gene and Cell Therapy (ASGCT) neue pr\u00e4klinische Daten aus der Pipeline von RNA-Editing-Therapeutika des Unternehmens, einschlie\u00dflich des Leitkandidaten [&hellip;]<\/p>\n","protected":false},"author":5,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[59],"tags":[],"class_list":["post-5484","post","type-post","status-publish","format-standard","hentry","category-pressemitteilungen"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>AIRNA stellt auf der ASGCT-Jahrestagung neue pr\u00e4klinische Daten zur therapeutischen RNA-Editing-Pipeline vor &#8212; Airna<\/title>\n<meta name=\"description\" content=\"In-vivo-Daten unterst\u00fctzen ein potenzielles Best-in-Class-Profil f\u00fcr AIR-001, das derzeit in einer klinischen Phase-I-Studie zu\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/airna.com\/de\/news\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\/\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"AIRNA stellt auf der ASGCT-Jahrestagung neue pr\u00e4klinische Daten zur therapeutischen RNA-Editing-Pipeline vor &#8212; Airna\" \/>\n<meta property=\"og:description\" content=\"In-vivo-Daten unterst\u00fctzen ein potenzielles Best-in-Class-Profil f\u00fcr AIR-001, das derzeit in einer klinischen Phase-I-Studie zu\" \/>\n<meta property=\"og:url\" content=\"https:\/\/airna.com\/de\/news\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\/\" \/>\n<meta property=\"og:site_name\" content=\"Airna\" \/>\n<meta property=\"article:published_time\" content=\"2026-04-28T11:00:00+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-08-19T20:40:48+00:00\" \/>\n<meta name=\"author\" content=\"Christine Mackenzie\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Verfasst von\" \/>\n\t<meta name=\"twitter:data1\" content=\"Christine Mackenzie\" \/>\n\t<meta name=\"twitter:label2\" content=\"Gesch\u00e4tzte Lesezeit\" \/>\n\t<meta name=\"twitter:data2\" content=\"3\u00a0Minuten\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/news\\\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/news\\\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\\\/\"},\"author\":{\"name\":\"Christine Mackenzie\",\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/#\\\/schema\\\/person\\\/452a34e50b25eae59fec12d65e4326af\"},\"headline\":\"AIRNA stellt auf der ASGCT-Jahrestagung neue pr\u00e4klinische Daten zur therapeutischen RNA-Editing-Pipeline vor\",\"datePublished\":\"2026-04-28T11:00:00+00:00\",\"dateModified\":\"2026-08-19T20:40:48+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/news\\\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\\\/\"},\"wordCount\":546,\"publisher\":{\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/#organization\"},\"articleSection\":[\"Pressemitteilungen\"],\"inLanguage\":\"de\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/news\\\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\\\/\",\"url\":\"https:\\\/\\\/airna.com\\\/de\\\/news\\\/airna-to-present-new-preclinical-data-on-rna-editing-therapeutic-pipeline-at-asgct-annual-meeting\\\/\",\"name\":\"AIRNA stellt auf der ASGCT-Jahrestagung neue pr\u00e4klinische Daten zur therapeutischen RNA-Editing-Pipeline vor &#8212; 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