Arnatar Therapeutics Announces Upcoming Presentations at AASLD The Liver Meeting® 2025 and ASN Kidney Week 2025

SAN DIEGO, Oct. 20, 2025 (GLOBE NEWSWIRE) -- Arnatar Therapeutics, a biotechnology company pioneering RNA-based therapies for severe and underserved diseases, today announced that new preclinical data from its ACT-UP1 platform will be presented at two upcoming scientific conferences in November 2025. The data will be featured at the American Society of Nephrology (ASN) Kidney Week in Houston, Texas (November 5-9, 2025), and the American Association for the Study of Liver Diseases (AASLD), The Liver Meeting® in Washington, D.C. (November 7-11, 2025).

“These upcoming presentations underscore the breadth and versatility of our ACT-UP1 platform,” said Xuehai Liang, Ph.D., Chief Executive Officer of Arnatar Therapeutics. “By demonstrating the ability to safely upregulate critical proteins like HNF4α, Jagged-1, and PC1, we are highlighting the transformative potential of antisense upregulation to address the root causes of serious liver and kidney diseases that currently have limited treatment options. We are excited to share these data with the scientific community as we continue to advance novel RNA medicines for patients in need.”

ASN Kidney Week 2025

Oral Presentation

Title: ASO-mediated PC1 Upregulation by Enhancing PKD1 Translation Has the Potential to Treat ADPKD
Presentation Type: Oral
Abstract #: FR-OR054
Date/Location: 4:30-6:00 pm CST on November 7, 2025, Houston, Texas

Summary: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a progressive genetic disorder characterized by cyst growth and kidney failure, with limited treatment options that do not address the root cause. Arnatar will present new preclinical data on ART5, a novel antisense oligonucleotide (ASO) developed using the company’s proprietary ACT-UP1 platform, designed to directly enhance translation of PKD1 mRNA and increase polycystin-1 (PC1) protein expression levels. ART5 demonstrated up to a two-fold increase in PC1 expression across human, mouse, and non-human primate cells. In ADPKD patient-derived cells, PC1 upregulation was associated with functional improvements, including reduced cAMP levels, decreased cyst formation, and lower cell proliferation—key hallmarks of disease modification. In vivo, subcutaneous dosing of ART5 led to durable increases in kidney PC1 protein in mice, supporting the potential for convenient monthly or less frequent administration. ART5 also demonstrated good tolerability and high specificity, with no detectable off-target effects. Collectively, these findings position ART5 as a highly specific, long-acting, and potentially transformative therapeutic candidate for ADPKD.

AASLD The Liver Meeting® 2025

Poster Presentation #1

Title: Antisense-Mediated Upregulation of HNF4α as a Potential Novel Therapeutic Strategy for Liver Fibrosis
Presentation Type: Poster
Poster # 4024
Date/Location: 8:00 am-5:00 pm EST November 10, 2025, Washington, D.C.

Summary: Hepatocyte nuclear factor 4 alpha (HNF4α) is a master regulator of hepatic gene expression and is essential for maintaining metabolic homeostasis. Reduced HNF4α expression has been linked to metabolic disorders and liver fibrosis. While mRNA and AAV delivery approaches have demonstrated proof-of-concept benefit in preclinical models, there remains a need for alternative strategies to safely and effectively restore HNF4α levels. Arnatar’s preclinical data demonstrate the potential of antisense oligonucleotides (ASOs) from its ACT-UP1 platform to upregulate HNF4α protein expression as a novel therapeutic strategy for liver fibrosis.

Poster Presentation #2

Title: Antisense Oligonucleotide-Mediated Upregulation of Jagged-1 Expression Ameliorates Disease Phenotypes in an ALGS Mouse Model
Presentation Type: Poster
Poster # 4591
Date/Location: 8:00am-5:00pm EST November 10, 2025, Washington, D.C.

Summary: Alagille Syndrome (ALGS) is a rare, autosomal dominant disorder primarily caused by JAG1 haploinsufficiency, leading to bile duct paucity, cholestasis, and progressive liver dysfunction. Arnatar’s ACT-UP1–engineered ASO candidate ART4 directly enhances JAG1 protein translation. In patient-derived cells and across multiple species, ART4 increased JAG1 protein by 40–80% (1.4–1.7-fold in ALGS cells) with no detectable off-target effects. In Jag1+/– neonatal mice, subcutaneous administration of ART4 restored bile duct morphogenesis, improved growth, and normalized liver function biomarkers, including bile acids, bilirubin, ALT, and AST. Enhanced expression was observed up to 60 days, and ART4 was well-tolerated in both rodents and non-human primates. These results support ART4 as a first-in-class, disease-modifying therapy for ALGS by directly addressing its genetic cause.

About Arnatar Therapeutics

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 new therapeutic possibilities for previously untreatable conditions. The company’s pipeline 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 is committed to transforming RNA innovation into life-changing, programmable medicines for patients worldwide.

Investor Contact:

Chris Nardo
LifeSci Advisors, LLC
cnardo@lifesciadvisors.com
646-517-2499

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