Korro Bio (KRRO) Chardan's 8th Annual Genetic Medicines Conference summary

Company overview and platform strategy

• Founded five years ago, raised $350 million, and went public last year; cash runway extends into 2026.

• Focuses exclusively on RNA editing using oligonucleotides to co-opt the ADAR enzyme for both rare and common diseases.

• Platform enables both repair of pathogenic mutations and modulation of wild-type proteins, expanding target opportunities.

• Recent learnings from lead program and platform design have accelerated development and attracted major pharma interest.

Lead program: Alpha-1 antitrypsin disease

• Lead candidate targets alpha-1 antitrypsin disease, addressing both liver and lung manifestations by repairing a single point mutation.

• Preclinical data show robust in vitro editing, no off-target effects, and strong efficacy in transgenic mouse models.

• Early clinical studies will likely use ZZ patients to demonstrate mechanism and efficacy.

• Regulatory filing for the lead program is planned by the end of the year, with preparations on track.

• LNP delivery was chosen over GalNAc to maximize alpha-1 levels and ensure efficacy during acute phase responses.

Strategic partnership and pipeline expansion

• Entered a $530 million deal with Novo Nordisk for two undisclosed cardiometabolic targets, with $10 million upfront and full R&D reimbursement.

• Partnership enables expansion into large, common diseases and provides non-dilutive funding without shifting focus from core liver and CNS programs.

• Novo Nordisk selected RNA editing for targets uniquely suited to this modality, aiming to address disease biology beyond monogenic disorders.

• Cash position of $155 million as of Q2, with runway into late 2026, not including additional partnership funds.