Aptadir Therapeutics

Aptadir Therapeutics is a preclinical biotechnology company developing a new class of RNA therapeutics for genetic disorders and oncology. Its therapeutic focus includes underserved conditions such as Fragile X Syndrome and Myelodysplastic Syndrome. Its platform is based on DNMTs Interacting RNAs (DiRs), a novel class of RNA inhibitors designed to reactivate genes silenced through aberrant DNA methylation. Aptadir’s lead programme is focused on Fragile X Syndrome, which is caused by silencing of the FMR1 gene on the X chromosome and the resulting loss or significant reduction of FMRP, a protein essential for normal brain development and function.