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Tfr2-ASO
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Tfr2-ASO is an antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals in collaboration with the IRCCS San Raffaele Scientific Institute. It is designed to target transferrin receptor 2 (TFR2), a protein expressed in hepatocytes and erythroid cells that plays a key role in regulating iron homeostasis and erythropoiesis. In hepatocytes, TFR2 activates the transcription of hepcidin, while in erythroid cells, it interacts with the erythropoietin (EPO) receptor to modulate EPO sensitivity. By downregulating TFR2 expression, Tfr2-ASO aims to increase circulating iron levels and enhance erythropoiesis. It has been investigated in preclinical research for the treatment of anemia associated with chronic kidney disease (CKD) and beta-thalassemia.
- Other names
- TFR2-ASOTFR-2-ASOTFR 2-ASOanti-Tfr2 antisense oligonucleotideanti-Tfr-2 antisense oligonucleotideanti-Tfr 2 antisense oligonucleotideTfr2 antisense oligonucleotideTfr-2 antisense oligonucleotideTfr 2 antisense oligonucleotide
02
Targets
TFR2 (Transferrin receptor 2)
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