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tonlamarsen
- Development stage
- Phase 2
- Lead developer
- Kardigan
- Modality
- 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
- Administration
- Subcutaneous
01
Overview
Tonlamarsen is an investigational antisense oligonucleotide designed to treat hypertension by targeting the underlying genetic cause of elevated blood pressure. It operates as a ligand-conjugated antisense oligonucleotide (LICA), specifically inhibiting the expression of angiotensinogen in the liver through RNA interference mechanisms. By binding to angiotensinogen mRNA and promoting its degradation via RNase H1-dependent cleavage, tonlamarsen reduces circulating levels of angiotensinogen—the most upstream component of the renin-angiotensin-aldosterone system (RAAS). This approach aims to provide more complete and sustained inhibition of RAAS activity compared to traditional antihypertensive therapies and may offer advantages in patients with resistant hypertension[3][4][6][8]. Tonlamarsen is being developed by Ionis Pharmaceuticals and is currently in phase II clinical trials for treatment-resistant hypertension[3].
- Other names
- ION 904ION904ION-904AGT-2.5-LAGT2.5-LAGT 2.5-Ltonlamarsen sodium
02
Targets
AGT (O6-methylguanine-DNA methyltransferase)
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