Korro Bio (KRRO) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Technology platform and approach

• Utilizes endogenous ADAR enzyme to achieve precise RNA editing, enabling single alphabet changes in RNA for therapeutic purposes.

• OPERA platform enables transient, reversible, and highly specific A-to-I (read as G) edits in RNA, expanding the genetic medicine toolkit beyond knockdown approaches.

• Platform is built internally, focusing on reducing risk by targeting areas with established delivery and chemistry, starting with rare liver diseases and expanding to CNS.

• RNA editing can address both rare Mendelian and chronic diseases caused by single nucleotide missense variants.

• Editing is achieved by delivering a synthetic oligonucleotide that binds specifically to the target RNA site, ensuring high specificity and efficiency.

Lead program: alpha-1 antitrypsin deficiency (AATD)

• Lead indication targets alpha-1 antitrypsin disease, caused by a single G to A mutation in the SERPINA1 gene, leading to protein misfolding and disease in liver and lung.

• KRRO-110, a single-stranded oligonucleotide encapsulated in a lipid nanoparticle, is delivered intravenously to the liver to correct the Z allele to the M allele.

• Preclinical studies in human cells, mice, and monkeys show high editing efficiency (>50%), high specificity, and no off-target effects.

• Achieved unprecedented protein levels in circulation and demonstrated histopathological resolution of liver disease in animal models.

• Regulatory filing planned for the second half of 2024, with clinical data expected in the second half of 2025.

Pipeline and future plans

• Pipeline includes programs for Parkinson's disease and ALS, leveraging the ability to repair pathogenic variants and modulate protein function.

• Focus remains on rare liver diseases initially, with plans to expand into other tissue types.

• Preclinical data supports translation across species, de-risking the approach for human trials.

• Strong financial position with runway into the second half of 2026, supporting completion of phase I/II study and advancement of additional programs.