Korro Bio (KRRO) Jefferies 2024 Global Healthcare Conference summary

Platform innovation and technology

• Developed OPERA, a platform enabling single-base RNA editing (A-to-I) using oligonucleotides and the endogenous ADAR enzyme.

• Focused on transient, reversible RNA edits to address both rare and prevalent diseases, aiming for commercial feasibility and patient impact.

• Utilizes computational methods and novel chemistries to optimize oligonucleotide design and delivery for high efficiency and specificity.

• Holds over 32 patent portfolios, supporting freedom to operate and broad application potential.

• Demonstrated ability to edit genes in both liver and neuronal cells, with modifiable efficiency based on chemistry.

Pipeline and lead asset progress

• Lead candidate KRRO-110 targets alpha-1 antitrypsin deficiency by repairing a pathogenic G-to-A variant in the SERPINA1 gene.

• Regulatory filing for KRRO-110 planned by end of this year, with clinical data expected in the second half of next year.

• Preclinical studies in humanized mice and monkeys show high editing efficiency (up to 60%) and restoration of normal protein levels.

• Demonstrated translation of efficacy from mice to monkeys, supporting confidence in human clinical outcomes.

• Sufficient capital secured to fund operations through late 2026.

Differentiation and safety profile

• Approach enables editing of any adenosine in the transcriptome, allowing for both repair and de novo protein creation.

• High specificity achieved with minimal off-target effects, validated by in vitro and in vivo studies.

• No depletion of endogenous ADAR activity observed, supporting chronic administration.

• Safety studies in mice and monkeys show favorable tolerability, outperforming approved RNAi therapies.

• Fit-for-purpose delivery strategies (GalNAc, LNP) enable high editing efficiency across tissues.