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SPC4955
- Development stage
- Phase 1
- Lead developer
- Roche
- 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, Small Molecules
- Administration
- Subcutaneous
01
Overview
SPC4955 is an investigational antisense oligonucleotide drug developed using the Locked Nucleic Acid (LNA) platform. It is designed to inhibit the synthesis of apolipoprotein B (apoB) mRNA, thereby reducing the production and export of low-density lipoprotein cholesterol (LDL-C) in the liver. By targeting apoB, SPC4955 aims to lower plasma levels of LDL-C and triglycerides, which are key risk factors for cardiovascular disease. The drug was advanced into Phase 1 clinical trials for subcutaneous administration in healthy volunteers to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics. The primary indication under investigation was hypercholesterolemia.
02
Targets
APOB (Apolipoprotein B)
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