Korro Bio (KRRO) Corporate presentation summary

Clinical progress and strategic pivots

• RNA editing demonstrated in AATD patients, with functional M-AAT produced and a tolerable safety profile observed in the KRRO-110 Phase 1/2a study.

• Single-dose KRRO-110 did not reach projected protein levels; multi-dose regimens are under consideration.

• Strategic shift to GalNAc-conjugated delivery for AATD, with a development candidate expected in H1 2026.

• KRRO-121 nominated for hyperammonemia, targeting a large market in urea cycle disorders and hepatic encephalopathy, with regulatory filing anticipated in H2 2026.

• Cash runway extends into H2 2027, with $102.5 million in cash and equivalents as of September 30, 2025.

Technology and platform overview

• RNA editing platform enables transient A-to-I edits using oligonucleotides and endogenous ADAR enzymes.

• Platform allows for gene correction, de novo protein generation, and modulation of gene expression.

• OPERA approach integrates ADAR biology, delivery expertise, machine learning, and chemistry for efficient candidate discovery.

• Delivery modalities include LNP (IV) and GalNAc (SC), targeting multiple cell types and indications.

Pipeline and market opportunities

• Pipeline includes KRRO-110 for AATD (Phase 1/2a), KRRO-121 for hyperammonemia (preclinical), and additional programs for ALS, pain, and cardiometabolic diseases.

• GalNAc-conjugated constructs show higher potency and align with strategic priorities for liver indications.

• Market opportunities estimated at $3B for AATD and $2B+ for hyperammonemia-related indications in the US.

• Collaboration with Novo Nordisk on up to two cardiometabolic targets, currently on a 12-month pause as of November 2025.