Arnatar Therapeutics Publishes Key Patents Underpinning ACT-UP1, a First-in-Class Protein Upregulation Platform
SAN DIEGO, CA, Sep. 8, 2025 -- Arnatar Therapeutics, a biotechnology company pioneering RNA-based therapies for severe and underserved diseases, today announced the publication of foundational patent applications covering ACT-UP1, a novel class of antisense compounds designed to upregulate protein expression. These applications cover ACT-UP1’s unique mechanism of action, composition, and broad therapeutic applications.
“Publication of these applications is an important milestone for Arnatar,” said Xuehai Liang, Ph.D., Chief ExecutiveOfficer of Arnatar Therapeutics. “ACT-UP1 is a novel platform with the potential to transform the treatment of diseases caused by haploinsufficiency and protein-deficiency.”
ACT-UP1 compounds represent a first-in-class approach to increase protein expression by hybridizing directly to target mRNAs and recruiting translation-related proteins to enhance translation. Unlike other strategies, ACT-UP1:
• Does not depend on inhibitory mRNA elements or co-delivery of exogenous proteins
• Enables upregulation across a broader range of genes
• Can theoretically boost any protein of interest
The compounds are short, chemically modified oligonucleotides that efficiently enter cells, demonstrate increased durability, and support less frequent dosing. Preclinical data demonstrated protein expression increases of up to approximately 130-200%.
Arnatar is advancing ACT-UP1 in multiple therapeutic areas, including Alagille Syndrome (ALGS) and AutosomalDominant Polycystic Kidney Disease (ADPKD).
About ArnatarTherapeutics
Arnatar Therapeutics is a clinical-stage biotechnology company redefining the possibilities of RNA medicine. With its proprietary DARGER™ platform, Arnatar uniquely integrates best-in-class siRNA gene silencing with first-in-class antisense oligonucleotides (ASOs) that upregulate protein expression. This dual-modality platform empowers Arnatar to develop RNA medicines that either silence harmful disease drivers through siRNA or restore essential protein function through up-regulating ASOs, opening newtherapeutic possibilities for previously untreatable conditions. The company’spipeline spans across cardiometabolic, liver, kidney, and central nervous system diseases, targeting areas of high unmet medical need. Founded by leaders in RNA therapeutics and backed by leading biotech investors, Arnatar iscommitted to transforming RNA innovation into life-changing, programmablemedicines for patients worldwide.
Investor Contact:
Chris Nardo
LifeSci Advisors, LLC
cnardo@lifesciadvisors.com
646-517-2499