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Sapablursen · ISIS-702843

Oligonucleotide (Antisense oligonucleotide) · Gene silencing: TMPRSS6 · developed by Ionis Pharmaceuticals

Phase 2 Most advanced trial (none active)

IONS Developer

1 Registered trials

What is Sapablursen?

Sapablursen is an antisense oligonucleotide targeting TMPRSS6 mRNA, conjugated to N-acetylgalactosamine for liver-targeted delivery. It blocks TMPRSS6 translation, preventing suppression of BMP-SMAD signaling and increasing hepcidin levels to regulate iron homeostasis and stimulate erythropoiesis. It is studied in polycythemia vera and iron overload disorders.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: ISIS-702843, IONIS-TMPRSS6-LRx
Drug type: Oligonucleotide (Antisense oligonucleotide)
Targets: TMPRSS6
Mechanism: Gene silencing: TMPRSS6
Pathways: BMP-SMAD signaling, hepcidin regulation, iron homeostasis, erythropoiesis
Therapy area: Oncology
Developer: Ionis Pharmaceuticals, Inc. (IONS)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 2
UNII: DXS4HJC32N

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT05143957	Phase 2	Phlebotomy Dependent Polycythemia Vera	Completed	IONS

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Phlebotomy Dependent Polycythemia Vera

More from Ionis Pharmaceuticals

Donidalorsen FDA approved Olezarsen FDA approved Zilganersen FDA approved BIIB094 Phase 1 ION224 Phase 2 ION269 Phase 1 ION337 Phase 1/2 ION356 Phase 1 ION363 Phase 3 ION440 Phase 1/2 ION464 Phase 1 ION717 Phase 1/2

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the IONS page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z