Tfr-DE3At

Tfr-DE3At is a chimeric RNA aptamer designed for the treatment of sickle cell disease (SCD). It is composed of a transferrin receptor (Tfr)-targeting aptamer linked to a truncated version of the anti-polymerizing RNA aptamer DE3A, known as DE3At. The Tfr-targeting component is intended to facilitate the internalization of the therapeutic molecule into reticulocytes and mature sickled red blood cells (RBCs) by binding to the transferrin receptor, which is highly expressed on these cell types. Once inside the cell, the DE3At component binds specifically to sickle hemoglobin (HbS), inhibiting its polymerization under deoxygenated conditions. This dual-action mechanism aims to reduce RBC sickling and delay polymer formation, addressing the underlying cause of SCD symptoms and complications. Preclinical data have demonstrated that Tfr-DE3At can successfully cross cellular membranes and reduce the sickling of mature RBCs in microfluidic assays.

Other names

Tfr-DE3At chimeric RNA aptamerTfr-DE-3At chimeric RNA aptamerTfr-DE 3At chimeric RNA aptamer