Portfolio News
SUZHOU, CHINA; SAN DIEGO, CA, Sep. 15, 2025 –Arnatar Therapeutics, a biotechnology company pioneering RNA-based therapies for severe and underserved diseases, today announced that the first patient has been dosed with ART104, a first-in-class antisense oligonucleotide (ASO) drug designed to treat Alagille Syndrome (ALGS) at its genetic root cause. No drug related AEs was observed, ART104 demonstrated well tolerated safety profile.
The investigator-initiated trial (IIT) is being conducted by Prof. Jianshe Wang at the Children’s Hospital of Fudan University, a globally recognized leader in ALGS research and care. The study will evaluate the safety, pharmacokinetics/pharmacodynamics (PK/PD), and efficacy of ART104 in patients living with ALGS.
“Dosing the first patient with ART104 is an important milestone not only for Arnatar but also for the ALGS community,” said Dr. Yanfeng Wang, COO and Head of Development. “By directly addressing Jag1 haploinsufficiency, ART104 has the potential to fundamentally change outcomes for patients and families living with this devastating disease.”
ART104: First-in-Class Approach to Restoring Jag1
ART104 is powered by Arnatar’s proprietary ACT-UP1 technology and is the first ASO therapeutic designed to upregulate Jag1 protein. Jag1 haploinsufficiency is recognized as the major cause of ALGS, a devastating multi system disease characterized by bile duct paucity and liver dysfunction.
In preclinical studies, ART104 demonstrated:
• Significant restoration of liver bile ducts in disease animal models
• Robust tolerability profile across multiple studies
ART104 has received both Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (PRDD) from the U.S. Food and Drug Administration (FDA), underscoring its potential as a transformative therapy.
Aiming to Change the Standard of Care
If successful, ART104 could offer the first efficient therapeutic approach targeting the underlying cause of ALGS, moving beyond symptomatic care to a disease-modifying treatment.
