ART5 is Arnatar’s antisense oligonucleotide (ASO) program targeting autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disorder, affecting over 10 million people globally. Approximately 85% of diagnosed ADPKD cases are caused by mutations in the PKD1 gene, leading to reduced expression of polycystin-1 (PC1), a key protein involved in maintaining tubular structure and kidney function. ART5 is designed to upregulate endogenous PKD1 expression, aiming to restore PC1 function and reduce the growth of cysts that characterizes disease progression.
Utilizing Arnatar’s ACT-UP1 platform, ART5 enhances translation of PKD1 mRNA without altering the gene itself. In preclinical models, ART5 has demonstrated the ability to increase PC1 protein levels and reduce cyst burden in a dose-dependent manner. It is administered subcutaneously and has shown favorable tolerability in vivo.
Given the current lack of effective treatments that directly address the underlying genetic cause of ADPKD, ART5 represents a novel approach that may shift the treatment paradigm toward disease modification. Development efforts are focused on advancing ART5 toward clinical studies, with a goal of evaluating safety, pharmacokinetics, and initial biological activity in patients with early-stage ADPKD.
ART5 is Arnatar’s antisense oligonucleotide (ASO) program targeting autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disorder, affecting over 10 million people globally. Approximately 85% of diagnosed ADPKD cases are caused by mutations in the PKD1 gene, leading to reduced expression of polycystin-1 (PC1), a key protein involved in maintaining tubular structure and kidney function. ART5 is designed to upregulate endogenous PKD1 expression, aiming to restore PC1 function and reduce the growth of cysts that characterizes disease progression.
Utilizing Arnatar’s ACT-UP1 platform, ART5 enhances translation of PKD1 mRNA without altering the gene itself. In preclinical models, ART5 has demonstrated the ability to increase PC1 protein levels and reduce cyst burden in a dose-dependent manner. It is administered subcutaneously and has shown favorable tolerability in vivo.
Given the current lack of effective treatments that directly address the underlying genetic cause of ADPKD, ART5 represents a novel approach that may shift the treatment paradigm toward disease modification. Development efforts are focused on advancing ART5 toward clinical studies, with a goal of evaluating safety, pharmacokinetics, and initial biological activity in patients with early-stage ADPKD.
