Korro Bio (KRRO) Jefferies London Healthcare Conference 2024 summary

Key platform and pipeline highlights

• Focuses on oligonucleotide-based RNA editing, converting adenosine to inosine for therapeutic effect.

• Lead program KRRO-110 targets Alpha-1 Antitrypsin Deficiency, with first dosing anticipated in Q1 2025.

• Pipeline expansion planned over the next few years, leveraging platform versatility.

• Collaboration with Novo Nordisk announced for up to two cardiometabolic targets, providing additional capital and milestones.

• Cash runway extends into the second half of 2026, supporting lead and additional programs.

Scientific and operational strengths

• Team and board have extensive experience in oligonucleotide drug development and gene editing.

• Platform leverages deep ADAR biology knowledge, oligonucleotide chemistry, and established delivery methods.

• Uses fit-for-purpose delivery, including GalNAc conjugates and lipid nanoparticles, tailored to indication needs.

• Machine learning aids in compound design, reducing risk and optimizing candidates.

Lead program: KRRO-110 for Alpha-1 Antitrypsin Deficiency

• Targets a G2A mutation in the SERPINA1 gene, aiming to restore normal protein levels.

• Delivered via IV lipid nanoparticle, with >90% liver targeting.

• Preclinical models show >60% editing and significant increases in functional M protein.

• Goal is to achieve at least 50% editing, mimicking the MZ phenotype for clinical benefit.

• Regulatory filing in Australia completed; first-in-human study to start in Q1 2025, focusing on safety and efficacy.