{"id":5464,"date":"2026-04-28T11:00:00","date_gmt":"2026-04-28T11:00:00","guid":{"rendered":"https:\/\/airna.com\/?p=5464"},"modified":"2026-04-28T11:06:05","modified_gmt":"2026-04-28T11:06:05","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 to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting"},"content":{"rendered":"\n<ul class=\"wp-block-list\">\n<li><em>In vivo data support potential best-in-class profile for AIR-001, currently in Phase 1 clinical trial for alpha-1 antitrypsin deficiency (AATD)<\/em><\/li>\n\n\n\n<li><em>Data from cardiometabolic portfolio demonstrate promise of AIRNA\u2019s approach to introduce beneficial variants<\/em><\/li>\n<\/ul>\n\n\n\n<p><strong>CAMBRIDGE, Mass., April 28, 2026<\/strong> \u2013 <a href=\"https:\/\/airna.com\/\">AIRNA<\/a>, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common conditions, will present new preclinical data from the company\u2019s pipeline of RNA-editing therapeutics, including lead candidate AIR-001 and the company\u2019s cardiometabolic portfolio, at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). <\/p>\n\n\n\n<p>In an oral presentation, AIRNA will present the first <em>in vivo<\/em> data on AIR-001, AIRNA\u2019s lead RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD). AIR-001 is a subcutaneously delivered GalNAc oligonucleotide designed to correct the most common disease-causing mutation of AATD (PiZ) at the RNA level by recruiting endogenous ADAR enzymes to precisely edit&nbsp;<em>SERPINA1<\/em>&nbsp;mRNA. AIR-001 is currently being evaluated in the Phase 1 RepAIR1 clinical trial (<a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07431112\">NCT07431112<\/a>).<\/p>\n\n\n\n<p>Additionally, in a poster at ASGCT, the company will share preclinical proof-of-concept studies utilizing RNA editing to introduce naturally occurring protective variants in the transcripts of two genes implicated in cardiovascular disease: low-density lipoprotein receptor (LDLR) and apolipoprotein B (APOB). These findings highlight the breadth of AIRNA\u2019s RNA-editing platform, which has the potential to precisely introduce beneficial changes at the RNA level.<\/p>\n\n\n\n<p>Details of the presentations are as follows:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Oral Presentation Title:<\/strong>&nbsp;\u201cAIR-001: A highly potent and precise GalNAc-conjugated RNA base editing treatment for alpha-1 antitrypsin deficiency\u201d<\/li>\n\n\n\n<li><strong>Abstract Number:<\/strong>&nbsp; 77<\/li>\n\n\n\n<li><strong>Session:<\/strong>&nbsp;Translational Gene Therapy and Editing in Storage Disorders<\/li>\n\n\n\n<li><strong>Date\/Time:<\/strong>&nbsp;Tuesday, May 12, 11:15 \u2013 11:30am ET<\/li>\n\n\n\n<li><strong>Location:<\/strong>&nbsp;MCEC Room 258ABC<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Poster Presentation Title:<\/strong>&nbsp;\u201cRNA Editing: A Modality for Precise Introduction of Gain-of-Function Variants Identified by Human Genetics\u201d<\/li>\n\n\n\n<li><strong>Abstract Number:<\/strong> 1426<\/li>\n\n\n\n<li><strong>Session:<\/strong>&nbsp;Poster Reception<\/li>\n\n\n\n<li><strong>Date\/Time:<\/strong> Tuesday, May 12, 5 pm &#8211; 6:30 pm<\/li>\n\n\n\n<li><strong>Location:<\/strong> MCEC Exhibit and Poster Hall B2-C, Exhibit level<\/li>\n<\/ul>\n\n\n\n<p><strong>About AIR-001<\/strong><\/p>\n\n\n\n<p>AIR-001 is AIRNA\u2019s lead RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD), a genetic disease most commonly caused by the PiZ mutation in the <em>SERPINA1<\/em> gene. AIR-001 is designed to repair the underlying cause of disease and restore production of functional alpha-1 antitrypsin protein, with the goal of addressing both the lung and liver manifestations of AATD. Administered via subcutaneous injection, AIR-001 is designed for convenient, repeat dosing. AIR-001 has been granted Orphan Drug Designation for the treatment of alpha-1 antitrypsin deficiency from the U.S. Food and Drug Administration and is currently being evaluated in the Phase 1 RepAIR1 clinical trial (<a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07431112\">NCT07431112<\/a>).<\/p>\n\n\n\n<p><strong>About AIRNA<\/strong><\/p>\n\n\n\n<p>AIRNA is a clinical-stage company developing RNA-editing therapeutics designed to repair disease-causing genetic variants and introduce beneficial variants that promote human health. The company\u2019s proprietary platform is based on research by RNA-editing pioneers and co-founders Thorsten Stafforst (University of T\u00fcbingen) and Jin Billy Li (Stanford University). AIRNA is advancing a pipeline of RNA-editing therapeutics led by a product candidate for alpha-1 antitrypsin deficiency (AATD). AIRNA is headquartered in Cambridge, MA, with research operations in T\u00fcbingen, Germany. Learn more at <a href=\"https:\/\/airna.com\/\">https:\/\/airna.com\/<\/a>.<\/p>\n\n\n\n<p><strong>Media:<\/strong><\/p>\n\n\n\n<p>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\n\n<p><\/p>\n","protected":false},"excerpt":{"rendered":"<p>CAMBRIDGE, Mass., April 28, 2026 \u2013 AIRNA, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common conditions, will present new preclinical data from the company\u2019s pipeline of RNA-editing therapeutics, including lead candidate AIR-001 and the company\u2019s cardiometabolic portfolio, at the 2026 Annual Meeting of the American Society of [&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":[35],"tags":[],"class_list":["post-5464","post","type-post","status-publish","format-standard","hentry","category-press-releases"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>AIRNA to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting &#8212; Airna<\/title>\n<meta name=\"description\" content=\"In vivo data support potential best-in-class profile for AIR-001, currently in Phase 1 clinical trial for alpha-1 antitrypsin deficiency (AATD) Data from\" \/>\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 to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting &#8212; Airna\" \/>\n<meta property=\"og:description\" content=\"In vivo data support potential best-in-class profile for AIR-001, currently in Phase 1 clinical trial for alpha-1 antitrypsin deficiency (AATD) Data from\" \/>\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=\"2026-04-28T11:06:05+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 to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting\",\"datePublished\":\"2026-04-28T11:00:00+00:00\",\"dateModified\":\"2026-04-28T11:06:05+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\":531,\"publisher\":{\"@id\":\"https:\\\/\\\/airna.com\\\/de\\\/#organization\"},\"articleSection\":[\"Press Releases\"],\"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 to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting &#8212; 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