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Korro Bio GalNAc-conjugated SERPINA1 RNA editing oligonucleotide

Development stage: Preclinical
Lead developer: Korro Bio
Modality: MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

Korro Bio is developing a GalNAc-conjugated RNA editing oligonucleotide targeting the SERPINA1 gene for the treatment of alpha-1 antitrypsin deficiency (AATD). Utilizing the proprietary OPERA (Oligonucleotide Promoted Editing of RNA) platform, the drug is designed to recruit endogenous ADAR (Adenosine Deaminase Acting on RNA) enzymes to site-specifically edit the pathogenic G-to-A mutation in SERPINA1 mRNA transcripts. The GalNAc conjugation enables targeted delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR). This approach aims to restore the production of functional alpha-1 antitrypsin protein and reduce the accumulation of misfolded protein in the liver, potentially addressing both the pulmonary and hepatic manifestations of AATD. This program is distinct from Korro Bio's KRRO-110, which utilizes lipid nanoparticle (LNP) delivery.

Other names: Korro Bio AATD GalNAc programGalNAc-conjugated RNA editing oligonucleotide (SERPINA1)

Targets

ASGPR (Asialoglycoprotein Receptor 1)ADAR (Adenosine deaminases acting on RNA (ADAR) family)Serpin family A member 1 PiZ-variant messenger RNA (SERPINA1 PiZ mRNA)

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