{"id":62789,"date":"2026-03-14T07:06:38","date_gmt":"2026-03-14T06:06:38","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/yoltech-therapeutics-receives-fda-clearance-to-initiate-phase-2-3-study-of-in-vivo-gene-editing-therapy-yolt-202-in-alpha-1-antitrypsin-deficiency-aatd\/"},"modified":"2026-03-14T07:06:38","modified_gmt":"2026-03-14T06:06:38","slug":"yoltech-therapeutics-receives-fda-clearance-to-initiate-phase-2-3-study-of-in-vivo-gene-editing-therapy-yolt-202-in-alpha-1-antitrypsin-deficiency-aatd","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/yoltech-therapeutics-receives-fda-clearance-to-initiate-phase-2-3-study-of-in-vivo-gene-editing-therapy-yolt-202-in-alpha-1-antitrypsin-deficiency-aatd\/","title":{"rendered":"YolTech Therapeutics Receives FDA Clearance to Initiate Phase 2\/3 Study of In Vivo Gene-Editing Therapy YOLT-202 in Alpha-1 Antitrypsin Deficiency (AATD)"},"content":{"rendered":"
\n

SHANGHAI–(BUSINESS WIRE)–YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene-editing therapies, today announced that the U.S. Food and Drug Administration (FDA)<\/b> has approved the Investigational New Drug (IND) application<\/strong> for YOLT-202<\/strong>, the company\u2019s investigational in vivo base-editing therapy<\/strong> for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)<\/strong>.<\/p>\n

\nThe FDA approval enables the initiation of an open-label, single-dose expansion Phase 2\/3 clinical study<\/strong> to evaluate the efficacy and safety of YOLT-202 in adult patients with Alpha-1 Antitrypsin Deficiency (AATD). The study is designed as a multiregional clinical trial (MRCT) to be conducted at clinical sites in the U.S. and other countries.<\/p>\n

\nYOLT-202 is currently being investigated in a first-in-human investigator-initiated trial (IIT) (NCT07193615) designed to evaluate the safety, tolerability, and preliminary efficacy of YOLT-202 in patients with AATD. As of the date of this release, two patients had been enrolled and completed dose. Following administration of YOLT-202, both patients showed rapid, robust and dose-dependent increases in AAT level as early as in Week 1. AAT levels in both patients reached above the protective threshold of 11 \u03bcM. Additionally, AAT levels increased to normal range (>20 \u03bcM) in the 45 mg dose group. These newly produced AAT proteins were both structurally corrected (M-AAT) and functional, with the proportion of corrected M-AAT increasing to >95% in the 45 mg dose group.<\/p>\n

\n\u201cThe FDA clearance, together with the encouraging early clinical data, highlights the transformative potential of YOLT-202 and our in vivo base-editing platform,\u201d stated Yuxuan Wu, M.D., Co-Founder and CEO of YolTech Therapeutics. \u201cWe are excited to advance this Phase 2\/3 study and bring a potential \u2018one and done\u2019 therapy to patients with Alpha-1 Antitrypsin Deficiency.\u201d<\/p>\n

\nAbout YOLT-202<\/span><\/strong><\/p>\n

\nYOLT-202 is an in vivo<\/i> gene-editing therapy that corrects PiZ mutation to PiM for the treatment of AATD. Utilizing YolTech\u2019s proprietary adenine base editor, YOLT-202 is engineered to achieve on-target editing with minimal bystander activity. YOLT-202 has previously been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).<\/p>\n

\nAbout Alpha-1 Antitrypsin Deficiency (AATD)<\/span><\/strong><\/p>\n

\nAATD is an inherited, genetic, autosomal co-dominant disorder caused by mutations in the SERPINA1 gene, with the most frequent deficient variants coming from the Z (Glu342Lys) and S alleles (Glu264Val). The presence of Z alleles results in misfolding and polymerization of the AAT, leading to over 95% of severe AATD patients being PIZZ.<\/p>\n

\nAbout YolTech<\/span><\/b><\/p>\n

\nBuilt on HEPDONE\u2122 Novel Editor Platform and non-viral LNP technologies, YolTech Therapeutics is advancing in vivo gene-editing medicines with the potential for a one-time treatment that provides lifelong benefit. The company\u2019s expanding clinical pipeline addresses genetic, metabolic, cardiovascular, and autoimmune diseases, with early clinical results supporting the potential for durable and transformative outcomes.<\/p>\n

\nStay informed with the latest from YolTech Therapeutics:
\n
LinkedIn: linkedin.com\/company\/yoltech-therapeutics<\/a>
Website: www.yoltx.com<\/a><\/p>\n

Contacts<\/b> <\/p>\n

\ncompany name: YolTech Therapeutics
\n
media contact: Ally Yu
\n
email address: xiaolingyu@yoltx.com<\/a><\/strong><\/p>\n<\/div>\n","protected":false},"excerpt":{"rendered":"

SHANGHAI–(BUSINESS WIRE)–YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene-editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for YOLT-202, the company\u2019s investigational in vivo base-editing therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The FDA approval enables the initiation of … [Read more…]<\/a><\/span><\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[13],"tags":[],"class_list":{"0":"entry","1":"post","2":"publish","3":"author-business","4":"post-62789","6":"format-standard","7":"category-industry"},"yoast_head":"\nYolTech Therapeutics Receives FDA Clearance to Initiate Phase 2\/3 Study of In Vivo Gene-Editing Therapy YOLT-202 in Alpha-1 Antitrypsin Deficiency (AATD) - Pharma Trend<\/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:\/\/pharma-trend.com\/en\/yoltech-therapeutics-receives-fda-clearance-to-initiate-phase-2-3-study-of-in-vivo-gene-editing-therapy-yolt-202-in-alpha-1-antitrypsin-deficiency-aatd\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"YolTech Therapeutics Receives FDA Clearance to Initiate Phase 2\/3 Study of In Vivo Gene-Editing Therapy YOLT-202 in Alpha-1 Antitrypsin Deficiency (AATD) - Pharma Trend\" \/>\n<meta property=\"og:description\" content=\"SHANGHAI–(BUSINESS WIRE)–YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene-editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for YOLT-202, the company\u2019s investigational in vivo base-editing therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). 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