Arnatar Therapeutics Announces First-in-Human Dosing of ART104, a First-in-Class ASO Therapy for Alagille Syndrome, in Investigator-Initiated Trial

On September 15, Arnatar Therapeutics announced that its ART104 drug had completed the first patient dosing in an investigator-initiated clinical trial (IIT) on September 1, 2025, Beijing time. The patient experienced no discomfort throughout the process. The first follow-up visit after administration has been completed, and no drug-related side effects were found during the evaluation, indicating good safety.ART104 is a potential "first-in-class" antisense oligonucleotide (ASO) drug designed to treat Alagille syndrome (ALGS) by targeting its genetic root cause.

The IIT trial is led by Professor Wang Jianguo from the Children's Hospital of Fudan University. This study will preliminarily evaluate the safety, pharmacokinetics/pharmacodynamics (PK/PD), and efficacy of ART104 in ALGS patients.

"The completion of the first patient dosing in the ART104 trial is not only an important milestone for Arnatar, but also a significant advancement in the entire ALGS field," said Dr. Yanfeng Wang, Chief Operating Officer and Head of Drug Development at Arnatar Therapeutics.By directly addressing the issue of insufficient Jag1 protein levels, ART104 has the potential to fundamentally change the disease outcomes for ALGS patients and their families.”

ART104, based on Arnatar's self-developed ACT-UP1 technology, is the first ASO therapeutic drug aimed at upregulating Jag1 protein expression.Low levels of Jag1 protein due to insufficient Jag1 protein are considered the primary cause of ALGS, a severe multisystem disorder characterized by bile duct paucity and liver dysfunction.

ART104 is dedicated to changing the existing treatment standards and is expected to become an effective therapeutic drug targeting the root cause of ALGS.In preclinical studies, ART104 has demonstrated initial advantages, including significant restoration of liver bile duct development in disease animal models and showing good tolerance in multiple toxicology studies. ART104 has been granted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (PRDD) by the U.S. FDA.

Arnatar is a clinical-stage biotechnology company dedicated to redefining the possibilities of RNA medicine. Arnatar owns the self-developed DARGER™ technology platform, which includes optimized siRNA gene silencing technology and oligonucleotide (ASO) protein upregulation technology. This dual-technology platform enables Arnatar to develop various RNA-targeting drugs: it can silence harmful pathogenic factors through siRNA or restore key protein functions via upregulating ASOs, thus opening new therapeutic avenues for previously untreatable diseases. The company’s R&D pipeline covers cardiovascular metabolism, liver, kidney, and central nervous system diseases, targeting areas with high unmet medical needs.

References:
[1]ArnatarPress Release

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