Aptadir Ce-49

Aptadir Ce-49 is a first-in-class RNA therapeutic developed by Aptadir Therapeutics for the treatment of myelodysplastic syndrome (MDS). It is based on a novel class of RNA inhibitors known as DNMTs Interacting RNAs (DiRs), which are designed to block aberrant DNA methylation at the single-gene level. By inhibiting DNA methyltransferase 1 (DNMT1), these molecules can reactivate previously hypermethylated tumor suppressor genes, addressing one of the root causes of MDS and potentially reversing disease progression. The therapy uses a lipid nanoparticle (LNP) delivery system engineered to target antigens specific to myeloid cells, enhancing precision and minimizing off-target effects. Preclinical studies have shown that Aptadir Ce-49 is highly selective, stable, non-toxic, and more effective than current standard-of-care treatments in disease models[1][2][4].

Brand names

Aptadir Ce-49