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.
Urea Cycle Disorders / Hepatic Encephalopathy (KRRO-121)
De novo protein to inhibit degradation
Item
- We are developing a GalNAc-conjugated oligonucleotide that generates a de novo, stabilized variant of an enzyme involved in ammonia clearance.
Alpha-1 antitrypsin deficiency (GalNAc Program)
Repairing a pathogenic variant
Item
- We are developing a GalNAc-conjugated oligonucleotide targeting the SERPINA1 gene for the potential treatment of AATD.
AMPKγ1 / Longevity (Liver)
Allosteric Activator
Item
- We are developing a GalNAc-conjugated oligonucleotide to improve liver function and restore metabolic signaling by activating hepatic AMPKγ1.
Amyotrophic lateral sclerosis
De novo protein to disrupt aggregation
Item
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.