Drug intelligence / Profile preview

ATR-1086

Development stage
Preclinical
Lead developer
Atrium Therapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

ATR-1086 is a preclinical small interfering RNA (siRNA) therapeutic candidate developed by Atrium Therapeutics for the treatment of phospholamban (PLN) cardiomyopathy. PLN cardiomyopathy is a rare, autosomal dominant genetic disease characterized by progressive heart failure for which no disease-modifying therapies currently exist. ATR-1086 utilizes a targeted, non-viral RNA delivery platform designed to selectively deliver the siRNA payload directly to cardiac tissue. By targeting the underlying genetic drivers of the disease—specifically the phospholamban gene—the therapy aims to silence the expression of the pathogenic protein and address the root cause of the cardiomyopathy. Atrium Therapeutics, a spin-out from Avidity Biosciences following its acquisition by Novartis, is currently advancing the candidate through CMC preparation with plans to initiate IND-enabling studies in 2026.

02

Targets

PLN (Phospholamban)

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