ADARx Pharmaceuticals is pioneering an innovative RNA targeting platform designed to develop therapeutics for a variety of diseases, including genetic, cardiometabolic, and central nervous system (CNS) disorders. The company employs a modality-agnostic approach that encompasses RNA editing, degradation, and inhibition, utilizing modified oligonucleotides to effectively regulate gene expression. Their RNA editing technology allows for precise modifications at the single base level, leveraging the body's existing cellular machinery to create stable and efficient therapeutics. This platform is currently being applied in multiple active programs, with a lead program already in Phase Ib clinical studies.

In addition to RNA editing, ADARx has developed novel delivery technologies aimed at enhancing the therapeutic potential of their RNA-based treatments. Their liver delivery system utilizes GalNAc, a sugar molecule that facilitates targeted uptake of therapeutics by binding to receptors on liver cells, thereby improving the efficacy of treatments for liver-related conditions. Furthermore, the company is actively researching specific mechanisms for delivering therapeutics to the CNS, addressing a critical area of unmet medical need. With a robust intellectual property portfolio, ADARx is well-positioned to advance its diverse pipeline of RNA-targeting therapeutics across various disease indications.

United States

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

ADARx Pharmaceuticals

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