Korro Bio (KRRO) 44th Annual J.P. Morgan Healthcare Conference summary

Technology Overview and Platform Strategy

• Focuses on developing transformative medicines for rare and prevalent diseases by activating biological pathways using RNA editing, specifically converting adenosine to inosine in RNA to impact protein structure and function.

• Utilizes a modular delivery system, including GalNAc-conjugated and chemically modified oligonucleotides, for precise targeting of specific cell and tissue types.

• OPERA platform is built on understanding ADAR biology, oligonucleotide chemistry, delivery technologies, and machine learning for target identification and efficient candidate discovery.

• RNA editing enables both repair of pathogenic protein variants and creation of novel, stabilized proteins to modulate or restore biological pathways.

• Emphasizes transient, non-permanent interventions, differentiating from gene therapy and DNA editing.

Pipeline Highlights and Clinical Development

• Lead program KRRO-121 targets stabilization of an intracellular protein to reduce ammonia in multiple indications, with regulatory filing and clinical entry expected in the second half of the year and a workshop on Jan. 27, 2026.

• KRRO-121 aims to treat all urea cycle disorders and hepatic encephalopathy, addressing large unmet needs with a subcutaneous, infrequent dosing regimen and a market opportunity estimated at $1.5B–$2B+ in the US and EU/UK.

• Alpha-1 antitrypsin deficiency (AATD) program is advancing toward a development candidate, demonstrating >90% editing of SERPINA1 transcript in vivo using GalNAc delivery, with candidate nomination expected in H1 2026.

• Additional programs include AMPK gamma-1 for metabolic diseases, showing improved liver function and reduced body weight in obese mice, and a novel approach to ALS by restoring TDP-43 function, both with promising preclinical results.

• Terminated Korro 110 after suboptimal clinical data, but leveraged learnings to improve next-generation candidates and delivery methods.

Key Learnings, Safety, and Future Outlook

• RNA editing offers high specificity with minimal off-target effects, as shown in preclinical and clinical studies.

• Gal-conjugated oligonucleotides provide potent, durable, and infrequent dosing, with sub-nanomolar EC50 achieved in lead programs.

• Platform enables rapid development cycles, reducing time to candidate nomination for new targets.

• Focuses on indications where transient modulation is advantageous over permanent gene editing, especially for chronic diseases.

• Cash runway extends into the second half of 2027, supporting continued pipeline advancement and milestone achievement.