RNA Targeting Technology
Turning cutting-edge science into life-saving therapeutics
Our expanding pipeline of RNA targeting therapeutics is currently focused on diseases in three therapeutic areas: genetic, cardiometabolic, and central nervous system (CNS) diseases. We currently have 9 active programs in development. Our lead program is entering IND-enabling toxicology studies.
Leveraging the power of ADARx platform technology
Through our RNA editing approach, we can specifically target an RNA sequence and, utilizing existing cellular machinery, precisely edit a single base. Our novel design is compact and stable resulting in efficient precision therapeutics. Our first editing program is directed to the treatment of Alpha-1 Antitrypsin Deficiency.
RNA Degradation and Inhibition
Our RNA degradation and inhibition approaches utilize modified oligonucleotides that mimic the body's natural biological processes to regulate gene expression. Our novel designs are highly potent and durable. Our platform allows reproducibility and consistency with multiple mechanisms and a wide range of indications.
Our novel liver delivery technology harnesses the benefits of GalNAc while maximizing atom economy. GalNAc, or N-acetylgalactosamine, is a sugar molecule that recognizes and binds to the cell surface asialoglycoprotein receptor (ASGPR), which is highly expressed in the liver, enabling targeted uptake of therapeutics in the liver.
We are developing specific mechanisms for delivering our therapeutics to the central nervous system. We have multiple programs at research stage that utilize our CNS delivery technology.
Our intellectual property portfolio provides broad protection of our unique platform technology allowing us the freedom to develop therapeutics across a broad range of disease indications.