Korro Bio (KRRO) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and technology

• Focuses on RNA editing using endogenous ADAR enzymes to make single base changes, enabling targeted gene modulation.

• Lead program targets alpha-1 antitrypsin deficiency, with a regulatory filing expected by year-end, clinical data in late next year, and study completion in 2026.

• Platform uses chemically modified, precedented RNA, allowing for scalable manufacturing and established delivery methods.

• Recently announced a partnership with Novo Nordisk to develop two cardiometabolic targets, expanding reach to large patient populations.

Scientific and clinical insights

• RNA editing can address a broad range of targets by correcting single point mutations or modulating protein function, with initial focus on rare Mendelian diseases.

• Preclinical data in animal models show high levels of protein correction and favorable safety, with strong translation from rodents to non-human primates.

• Demonstrated ability to address both liver and lung phenotypes in alpha-1 antitrypsin deficiency models.

• Safety studies indicate a large therapeutic index, with no observed toxicity at high doses in monkeys.

• Dosing frequency is projected at every three weeks, pending further clinical data.

Competitive positioning and differentiation

• RNA editing offers titratable, reversible gene modulation, suitable for chronic diseases and large, diverse patient populations.

• Can target both homozygous and heterozygous individuals, unlike DNA editing, and allows for patient-specific dosing.

• Differentiates from siRNA and antisense by focusing on activation or modulation rather than knockdown.

• Delivery leverages established LNP technology, reducing development risk and enabling efficient translation to clinic.