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fesomersen (Ionis Pharmaceuticals)

Development stage
Phase 2
Lead developer
Ionis Pharmaceuticals
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

Fesomersen (IONIS-FXI-Rx) is a second-generation 2-O-methoxyethyl (2-MOE) modified antisense oligonucleotide (ASO) designed to inhibit the synthesis of coagulation Factor XI (FXI) in the liver. By binding to the messenger RNA (mRNA) of FXI, fesomersen triggers RNase H-mediated degradation, leading to a significant reduction in circulating FXI protein levels. Factor XI is a key component of the intrinsic pathway of the coagulation cascade, and its inhibition is hypothesized to decouple thrombosis from hemostasis, potentially offering antithrombotic efficacy with a lower risk of bleeding compared to traditional anticoagulants. Developed by Ionis Pharmaceuticals and formerly partnered with Bayer, fesomersen was primarily investigated for the prevention of thromboembolic events in patients with end-stage renal disease (ESRD) undergoing hemodialysis. Following Phase 2 clinical results, development was discontinued by Bayer, and Ionis has since focused on a ligand-conjugated version (IONIS-FXI-L_Rx) for improved potency.

Other names
Factor XI antisense oligonucleotide
02

Targets

F11 (Coagulation Factor XI)

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