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AVT-01 is a double-stranded decoy oligodeoxynucleotide (ODN) designed to selectively inhibit the transcription factor STAT-1 (Signal Transducer and Activator of Transcription 1). Developed by Avontec GmbH (later known as DOLTHERA), it functions as a molecular sink that competitively binds to STAT-1, preventing the transcription factor from interacting with its endogenous promoter sequences on genomic DNA. This mechanism blocks the induction of pro-inflammatory genes involved in innate immunity and chronic inflammation. AVT-01 was primarily developed for the treatment of moderate persistent atopic asthma, where it reached Phase 2a clinical trials. In these studies, the drug was administered via inhalation to evaluate its efficacy in reducing airway hyperresponsiveness, as measured by changes in methacholine PC20. It is recognized as one of the first ODN-based drug candidates to enter clinical trials targeting a transcription factor in a specific and selective manner.
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