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opemalirsen

Development stage: Phase 1
Lead developer: AstraZeneca
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

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

Targets

APOL1 (Apolipoprotein L1)

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