Drug intelligence / Profile preview

opemalirsen

Development stage
Phase 1
Lead developer
AstraZeneca
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

Opemalirsen is an investigational antisense oligonucleotide (ASO) developed by AstraZeneca and Ionis Pharmaceuticals. It is designed to reduce the production of apolipoprotein L1 (APOL1) for the treatment of APOL1-mediated kidney disease (AMKD). The drug is administered via subcutaneous injection and is currently undergoing clinical trials to assess its efficacy, safety, and tolerability.

Other names
ION532ION-532ION 532IONIS-AZ5-2.5RxIONIS-AZ-5-2.5RxIONIS-AZ 5-2.5Rx
02

Targets

APOL1 (Apolipoprotein L1)

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