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IONIS-877864
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- 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
IONIS-877864 is a ligand-conjugated **antisense oligonucleotide (ASO)** designed to target and degrade mutant mRNA transcripts of the **DMPK** (dystrophia myotonica protein kinase) gene, which are responsible for the pathogenesis of **myotonic dystrophy type 1 (DM1)**. The drug employs a **C16-hexadecanoyl conjugation (C16-HA)** to enhance delivery to striated muscle tissue. This targeted approach results in significant knockdown of toxic mutant DMPK RNA, improvement in muscle strength, normalization of muscle stem cell populations, and reduction of muscle fibrosis in preclinical mouse models. Developed by Ionis Pharmaceuticals, preclinical studies highlight its efficacy and lack of observable toxicity in animal models.
- Other names
- C16-HA-ASOC-16-HA-ASOC 16-HA-ASO
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)
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