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sapablursen
- Development stage
- Phase 3
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
Sapablursen is a synthetic 2'-O-(2-methoxyethyl)-modified antisense oligonucleotide linked to a tri-antennary cluster of N-acetyl galactosamine (GalNAc3) sugars. It targets the messenger RNA of transmembrane protease, serine 6 (TMPRSS6), a negative regulator of hemojuvelin and thus an inhibitor of hepcidin production. By reducing TMPRSS6 expression, sapablursen increases endogenous hepcidin levels, which in turn decreases iron absorption and release from storage sites, normalizing iron levels and reducing red blood cell production. This mechanism is being investigated primarily for the treatment of polycythemia vera (PV), especially in patients who are phlebotomy-dependent. Sapablursen is administered as a subcutaneous injection every four weeks and has received Fast Track and Orphan Drug designation from the FDA for PV. The drug was originally developed by Ionis Pharmaceuticals and has been licensed globally to Ono Pharmaceutical for further development and commercialization[1][3][4][5][6][7][8].
- Other names
- sapablursen
02
Targets
TMPRSS6 (Transmembrane Serine Protease 6)
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