anti-IRF4 ASO

anti-IRF4 ASO (notably ION251) is an antisense oligonucleotide therapeutic candidate designed to selectively target and suppress the expression of Interferon Regulatory Factor 4 (IRF4). IRF4 is a transcription factor that serves as a critical master regulator for the survival and proliferation of multiple myeloma (MM) cells, but it has historically been considered undruggable due to its lack of traditional small-molecule binding pockets. Developed by Ionis Pharmaceuticals in collaboration with the Dana-Farber Cancer Institute, this ASO utilizes an RNase H1-dependent mechanism to induce the degradation of IRF4 mRNA. Preclinical data presented at ASH 2020 demonstrated that anti-IRF4 ASOs exhibit potent, dose-dependent anti-tumor activity across diverse multiple myeloma cell lines and show synergistic effects when combined with standard-of-care treatments such as proteasome inhibitors and immunomodulatory drugs (IMiDs).

Other names

IRF4 ASOIRF-4 ASOIRF 4 ASOanti-IRF4 antisense oligonucleotideanti-IRF-4 antisense oligonucleotideanti-IRF 4 antisense oligonucleotide