Korro Bio (KRRO) Corporate presentation summary

Strategic vision and technology platform

• Focus on developing transformative genetic medicines for both rare and prevalent diseases using RNA editing without permanent DNA modification.

• Utilizes a modular delivery system with potential to target multiple cell types and leverages genetic insights for predictable biological outcomes.

• OPERA platform integrates ADAR biology, machine learning, and chemistry expertise to drive efficient RNA editing and drug design.

Pipeline and anticipated milestones

• Lead asset KRRO-121 targets hyperammonemia with a regulatory filing anticipated in H2 2026.

• Development candidate announcement for GalNAc-conjugated AATD construct expected in Q2 2026, and a third GalNAc-conjugated liver asset in H2 2026.

• Cash position of $157.1 million as of March 31, 2026, with runway into H2 2028 to support multiple milestones.

Scientific rationale and preclinical data

• KRRO-121 uses liver-specific GalNAc-oligo to stabilize glutamine synthetase (GS), enhancing ammonia clearance.

• Demonstrated stabilization of GS in human cell models and significant ammonia reduction in OTC- and CPS-1-deficient mice.

• Preclinical safety studies in non-human primates showed no adverse signals or off-target editing.