Pipeline
Our strategy
Korro Bio is focused on diseases where our platform, OPERA®, can provide a unique solution. We are targeting disease-causing sequences that would be responsive to the safe and targeted editing of RNA. Our current focus areas include the liver and central nervous system.
Program/Indication
Urea Cycle Disorders / Hepatic Encephalopathy (KRRO-121)
Concept
Stabilized de novo protein
- Discovery
- Preclinical
- Phase 1
- Phase 2
- Phase 3
Wholly Owned
yes
KRRO-121 is a GalNAc-conjugated RNA editing oligonucleotide designed to generate a stabilized, de novo of glutamine synthetase (GS) protein, a critical enzyme involved in ammonia clearance. This synthetic rescue approach is designed to augment ammonia clearance in hyperammonemia-driven diseases such as UCDs and HE.
Program/Indication
Alpha-1 Antitrypsin Deficiency (KRRO-111)
Concept
Repairing a pathogenic variant
- Discovery
- Preclinical
- Phase 1
- Phase 2
- Phase 3
Wholly Owned
yes
KRRO-111 is a GalNAc-conjugated RNA editing oligonucleotide designed to address the underlying genetic cause of AATD and provide a differentiated therapeutic option compared to existing treatments.
Following evolutions in our platform chemistry, KRRO-111 is a proprietary RNA editing oligonucleotide conjugated with GalNAc to be administered subcutaneously with specific delivery to liver hepatocytes, where they are designed to co-opt endogenous ADAR to edit a nucleoside in the SERPINA1 transcript and restore production of normal AAT.
Program/Indication
AMPKγ1 / Longevity (Liver)
Concept
Allosteric Activator
- Discovery
- Preclinical
- Phase 1
- Phase 2
- Phase 3
Wholly Owned
yes
AMPK, when activated, inhibits anabolic pathways like lipogenesis and protein synthesis, activates catabolic pathways including fatty acid oxidation and autophagy, and regulates glucose homeostasis by enhancing insulin sensitivity. The γ1 subunit—AMPKγ1—represents the optimal liver therapeutic target because of its hepatocyte enrichment.
We are developing proprietary oligonucleotides designed to activate AMPKγ1, with the goal of restoring metabolic status and improving liver function. This program utilizes GalNAc-conjugated delivery for subcutaneous administration targeting the liver, designed to avoid activating AMPK in the rest of the body.
Program/Indication
Amyotrophic Lateral Sclerosis
Concept
De novo protein to disrupt aggregation
- Discovery
- Preclinical
- Phase 1
- Phase 2
- Phase 3
Wholly Owned
yes
We are developing proprietary oligonucleotides targeting the mRNA for TAR DNA binding protein 43 (TDP-43), which is associated with the development of amyotrophic lateral sclerosis (ALS).
ALS is an adult-onset, progressive, and fatal neurodegenerative disorder that causes muscle weakness, paralysis, and ultimately death. The majority of ALS patients die from respiratory failure within three to five years after symptom onset, with a small percentage surviving beyond 10 years. Despite being classified as a rare disease by the FDA and EMA, ALS is considered one of the more common neurodegenerative diseases worldwide