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IONIS-885417

Development stage: Preclinical
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

Overview

IONIS-885417 is a ligand-conjugated antisense oligonucleotide (ASO) control developed by Ionis Pharmaceuticals. It features a palmitate hexylamine phosphodiester (C16-HA) ligand conjugated to a control oligonucleotide sequence utilizing constrained ethyl (cEt) chemistry. In preclinical research for Myotonic Dystrophy Type 1 (DM1), it serves as a negative control to evaluate the efficacy, tissue distribution, and safety of therapeutic C16-HA-conjugated ASOs targeting the human dystrophia myotonica protein kinase (hDMPK) gene. The C16-HA conjugation is a Ligand-Conjugated Antisense (LICA) technology designed to enhance ASO uptake in striated muscle and cardiac tissues by improving binding to serum proteins like albumin and targeting specific cell surface receptors.

Other names: C16-HA-ASO controlC-16-HA-ASO controlC 16-HA-ASO control

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