Korro Bio, Inc. · NASDAQ:KRRO
Korro Bio — Investor Presentation (August 2026)
26 slides from Korro Bio's investor presentation, August 2026
Slide 1: Corporate Presentation
March2026 August 2025Corporate Presentation 11
Slide 2: Forward-Looking Statement and Disclaimers
Forward-Looking Statement and Disclaimers Forward-Looking Statements Certain statements in this presentation may constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements include, but are not limited to,
Slide 3: Our Vision
Our Vision Developing Transformative Genetic Medicines for Rare and Highly Prevalent Diseases 3
Slide 4: Activating Biological Pathways
Activating Biological Pathways Editing RNA Without permanently modifying DNA Modular Delivery Potential to deliver to multiple cell types Learning from Genetics To support predictable biological impact 4
Slide 5: Positioned for Value Creation in 2026 and Beyond
Positioned for Value Creation in 2026 and Beyond KRRO-111 Development Candidate (DC) nominated for GalNAc- conjugated alpha-1 antitrypsin deficiency (AATD) program Regulatory approval to commence FIH clinical trial for KRRO-121 anticipated in H2 2026 DC expected for a 3rd GalNAc-conjugated liver ass
Slide 6: OPERA: Our Approach for RNA Editing to Generate Product Candidates
OPERA: Our Approach for RNA Editing to Generate Product Candidates Expertise in ADAR biology driving potency and translation Leveraging known mechanisms to Expertise in Chemistry derisk Delivery driving potency and drug designs Expertise in Machine Learning driving efficiency and Target ID 6
Slide 7: RNA Editing Enables Potential for High Impact in Range of Disease Areas
RNA Editing Enables Potential for High Impact in Range of Disease Areas DNA Pre-mRNA mRNA Protein TRANSCRIPTION PROCESSING TRANSLATION Edit a single A-to-I Edit a single A-to-I Current • Repair a G->A mutation to correct the protein Focus Modifying gene expression • Generate a de novo protein with p
Slide 8: Pipeline Programs Primarily Focused on Generation of De Novo Proteins
Pipeline Programs Primarily Focused on Generation of De Novo Proteins Repair the Protein Modulate Protein Function (Activate pathway) TRANSLATION TRANSLATION RNA with a Pathogenic mutant RNA Normal Protein missense protein TRANSLATION TRANSLATION Edited RNA Normal “corrected” Edited RNA De Novo Prot
Slide 9: Pipeline with Potential High-Value Programs and Anticipated Milestones
Pipeline with Potential High-Value Programs and Anticipated Milestones PROGRAM / PRECLINICAL CONCEPT DELIVERY DISCOVERY PHASE 1 PHASE 2 PHASE 3 INDICATION DEVELOPMENT KRRO-121 Stabilize Protein GalNAc (SC) GS Reg approval in 2H 2026 Hyperammonemia KRRO-111 Repair Pathogenic Variant GalNAc (SC) AAT A
Slide 10: KRRO-121: Hyperammonemia
KRRO-121: Hyperammonemia Synthetic Rescue 10
Slide 11: Plasma Ammonia Significantly Impacts Pathology Across Multiple Diseases
Plasma Ammonia Significantly Impacts Pathology Across Multiple Diseases Ammonia Pathology ▪ High ammonia leads to: ‒ Neurological impairment, potentially permanent Diet & metabolism Normal ‒ Frequent hospitalization Clearance Normal excretion through kidneys ‒ Highly restricted diet ‒ Elevated infec
Slide 12: Two Complementary Pathways for Ammonia Clearance: Urea Cycle and Glutamine Synthetase
Two Complementary Pathways for Ammonia Clearance: Urea Cycle and Glutamine Synthetase (GS) Urea Cycle Glutamine Synthetase Ammonia Glutamate Ammonia OTC CPS-1 ARG Urea Cycle ASS Urea Glutamine synthetase ASL Glutamine Expressed Expressed in many tissues, primarily in liver including liver, brain, an
Slide 13: KRRO-121 is a GalNAc-conjugated Oligonucleotide for Liver-specific RNA Editing of GS
KRRO-121 is a GalNAc-conjugated Oligonucleotide Designed for Liver- specific RNA Editing of GS to Enhance Ammonia Clearance Capacity Glutamine Drives Degradation of GS Our Approach Glutamate Ammonia GalNAc Glutamine synthetase Glutamine N synthetase Edit Site GS Degradation KRRO-121 Prevents degrada
Slide 14: KRRO-121 Stabilized GS in UCD-derived Human Cell Models
KRRO-121 Stabilized GS in UCD-derived Human Cell Models KRRO-121 Stabilized GS in OTC-Deficient iPSC-Derived Hepatocytes 3 non treated GS degradation Similar results 10 mM NH4Cl Fold Change to Vehicle in ASS1-deficient 2 ✱✱✱ iPSC-derived hepatocytes GS remained stable upon 1.0 1.0 oligo treatment 0.
Slide 15: Ammonia Reduction in OTC- and CPS-1-Deficient Mice Supports Pan-UCD Potential
Ammonia Reduction in OTC- and CPS-1-Deficient Mice Challenged with Ammonia Supports Pan-UCD Potential and Diet Liberalization OTCspf/ash CPS-1 ✱✱ 2000 p < 0.05 1500 Vehicle Vehicle Mouse-optimized oligo Mouse-optimized oligo Ammonia (µM) 1500 Ammonia (μM) 1000 1000 Fasted ammonia 500 ULN: 450 M ns
Slide 16: KRRO-121 Has Blockbuster Potential in Multiple Indications
KRRO-121 Has Blockbuster Potential in Multiple Indications Urea Cycle Disorders Hepatic (UCD) Encephalopathy (HE) Addressable 4,200 U.S. 1 80,000 U.S. 2 Patients 5,100 EU + UK1 150,000 EU + UK3 Market Opportunity $1.5B $2B+ Note: 1. Severe late-onset UCD patients; 2. Patients prescribed rifaximin +/
Slide 17: KRRO-121: A Potential First-in-class Treatment For Ammonia Control
KRRO-121: A Potential First-in-class Treatment For Ammonia Control Preclinical Activity Preclinical Safety Demonstrated Translation ▪ Pan-UCD potential impacting ▪ NHP: No adverse safety signals in ▪ Production of stable, de novo GS multiple UCD subtypes repeat QWx3 dose range finding tox protein wh
Slide 18: KRRO-111: Alpha-1 Antitrypsin Deficiency
KRRO-111: Alpha-1 Antitrypsin Deficiency Repair Pathogenic Variant 18
Slide 19: AATD: An Autosomal Recessive Disorder Leading to Liver and Lung Pathology
AATD: An Autosomal Recessive Disorder Leading to Liver and Lung Pathology M-AAT is secreted from the liver and Healthy transported to the lungs, protecting Genotype (MM) against inflammatory damage M-AAT ! Z-AAT aggregates in the liver, leading to: AATD • Liver damage from Z-AAT aggregates Genotype
Slide 20: Current Standard of Care Approved Based on Lower Limit of MZ Phenotype
Current Standard of Care Approved Based on Lower Limit of MZ Phenotype Serum Ranges Across AAT Genotypes 50 MM • The current standard of care is weekly = Median AAT for genotype intravenous AAT augmentation therapy 40 (60 mg/kg/week) Serum AAT levels (μM) 30 MZ • Augmentation therapy targets serum A
Slide 21: KRRO-111 Demonstrated >90% Editing and ~90% M-AAT Protein In Vivo
KRRO-111, a GalNAc-conjugated RNA Editing Oligo, Demonstrated >90% Editing and ~90% M-AAT Protein In Vivo GalNAc KRRO-111 SERPINA1 RNA Editing % M-AAT Protein 100 91.8 100 84.3 M-AAT Dosing: Q2Dx3 at 5 or 10 mg/kg Proportion (%) Z-AAT 80 80 % Editing 60 60 NSG-PiZ Mouse Week 0 1 40 40 20 20 Day 7: s
Slide 22: KRRO-111 Nearly Eliminates Active Z Protein Production and Clears Aggregates
KRRO-111 Nearly Eliminates Active Z Protein Production and Drives Progressive Clearance of Pre-existing Aggregates In Vivo Elimination of Active Z-AAT Protein Production and Progressive Clearance of Pre-existing Aggregates Near-Complete Cessation Robust Reduction of Pre- Vehicle Control KRRO-111 of
Slide 23: Early Pipeline
Early Pipeline 23
Slide 24: Hepatic AMPK Activation Improved Liver Function in Obese Mice
GalNAc Hepatic AMPK Activation Improved Liver Function in Obese Mice Normalizing Liver Function Reduction in Body Weight Despite Similar Food Intake ALT AST 400 Vehicle 400 Vehicle % Body Weight Food Consumption Oligo 1 Oligo 1 105 8 230 Vehicle Food intake (g/day) 300 300 Arrows indicate Oligo 1
Slide 25: De Novo TDP-43 Reduced Mis-splicing and Cytosolic Mis-localization in iPSC Motor Neurons
De Novo TDP-43 Demonstrated Reduced Mis-splicing and Decreased Cytosolic Mis- localization in iPSC Motor Neurons Decreased Cytosolic Mis-localization of TDP-43 protein Reduced Mis-splicing: Maintaining POLDIP3, STMN2, and UNC13A TDP-43 DAPI Merge STMN2 Exon 1-2a Expression ✱✱✱ 20 Cell Stressor ✱✱✱✱
Slide 26: Positioned for Value Creation in 2026 and Beyond
Positioned for Value Creation in 2026 and Beyond KRRO-111 DC nominated for GalNAc-conjugated AATD program Regulatory approval to commence FIH clinical trial for KRRO-121 anticipated in H2 2026 DC expected for a 3rd GalNAc-conjugated liver asset in H2 2026 Cash runway into H2 ‘28 enabling multiple mi