Drug intelligence / Profile preview

EDP167

Development stage
Phase 2
Lead developer
Genor Biopharma
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

EDP167 is an antisense oligonucleotide (ASO) developed by Genor Biopharma that targets hepatic angiopoietin-like protein 3 (ANGPTL3) for the treatment of severe dyslipidemia and cardiovascular diseases. By inhibiting the expression of ANGPTL3 in the liver, the drug promotes the clearance of triglycerides and LDL cholesterol through a mechanism that is independent of the LDL receptor pathway. This makes it a promising therapeutic candidate for patients with homozygous familial hypercholesterolemia (HoFH), who often lack functional LDL receptors and do not respond adequately to conventional lipid-lowering agents like statins or PCSK9 inhibitors. EDP167 has completed Phase I testing in healthy volunteers and is currently being evaluated in Phase II clinical trials for adult patients with HoFH.

02

Targets

ANGPTL3 (Angiopoietin-like protein 3)

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