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ION775
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- 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
ION775 is an investigational antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of metabolic and endocrine disorders. It is designed to inhibit the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism that inhibits lipoprotein lipase and hepatic uptake of triglyceride-rich lipoproteins. By reducing APOC3 levels, ION775 aims to lower plasma triglycerides and mitigate cardiovascular risk. The drug is currently in Phase 1 clinical development and represents a next-generation candidate in Ionis's lipid-management portfolio, distinct from their more advanced APOC3-targeted assets like olezarsen.
02
Targets
APOC3 (Apolipoprotein C-III)
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