Slides
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1 1 March 2025 44th Annual J.P. Morgan Healthcare Conference Ram Aiyar President and Chief Executive Officer January 15, 2026
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2 2 Forward-Looking Statement and Disclaimers CONFIDENTIAL 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, express or implied statements regarding expectations, hopes, beliefs, intentions or strategies of Korro Bio, Inc. (Korro) regarding the future including, without limitation, express or implied statements regarding: Korro’s ability to activate a biological pathways with RNA editing; Korro’s ability to create value in 2026 and beyond; Korro’s cash runway and uses thereof; the timing of updates on Korro's assets; the timing of and ability to submit a regulatory filing for KRRO -121; the market opportunity for KRRO -121; the potential of Korro’s GalNAC-conjugated programs targeting the liver, including KRRO -121 and GalNAc delivery for AATD patients; and timing of nominating development candidates for Korro’s GalNAc-conjugated program for AATD and for a third GalNAC-conjugated liver asset. In addition, any statements that refer to projections, forecasts, or other characterizations of future events or circumstances, including any underlying assum ptions, are forward -looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “st rive,” “would,” “aim,” “target,” “commit,” and similar expressions may identify forward - looking statements, but the absence of these words does not mean that statement is not forward looking. Forward -looking statemen ts are based on current expectations and assumptions that, while considered reasonable are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to pred ict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including risks inhere nt in biopharmaceutical development; risks associated with pre -clinical studies and clinical studies; and other risks associated with obtaining regulatory approvals and protecting intellectual property; as wel l as risks associated with general economic conditions; the possibility that Korro may be adversely affected by other economic, business, and/or competitive factors; other risks and uncertainties indicated from time to time in Korro’s filings with the SEC, including Item 1A. “Risk Factors” in Korro’s most recent Quarterly Report on Form 10 -K or Form 10 -Q filed with the SEC, as such may be amended or supplemented by its other filing s with the SEC. Nothing in this presentation should be regarded as a representation by any person that the forward -looking statements set forth herein will be achieved or that any of the contemplated results of s uch forward -looking statements will be achieved. You should not place undue reliance on forward -looking statements in this presentation, which speak only as of the date they are made and are qualified in their ent irety by reference to the cautionary statements herein. Except as required by law, Korro does not undertake or accept any duty to release publicly any updates or revisions to any forward -looking statements to reflect any change in their expectations or in the events, conditions or circumstances on which any such statement is based. This presentation does not purport to summarize all of the conditions, risks and other attribute s of an investment in Korro. Industry and Market Data Certain information contained in this presentation relates to or is based on studies, publications, surveys and Korro’s own internal estimates and research. In this presentation, Korro relies on, and refers to, publicly available information and statistics regarding market participants in the sector in which Korro competes and other industry data. Any comparison of Korro to any other entity assumes the reliability of the information available to Korro. Korro obtained this information and statistics from third -party sources, including reports by market research firms and company filin gs. In addition, all of the market data included in this presentation involve a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. Finally, while Korro believes its internal research is reliable, such research has not been verified by any independent source and Korro has not independently verified the information. Trademarks This presentation may contain trademarks, service marks, trade names and copyrights of Korro or other third parties, which are the property of their respective owners. Solely for convenience, some of the trademarks, service marks, trade names and copyrights referred to in this presentation may be listed without the TM, SM © or ® symbols, but Korro will assert, to the fullest extent under applicable law, the rights of the applicable owners, if any, to its trademarks, service marks, trade names and copyrights.
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3 3 Our Vision Developing Transformative Genetic Medicines for Rare and Highly Prevalent Diseases
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4 4 Activating Biological Pathways Editing RNA Without permanently modifying DNA Learning from Genetics To support predictable biological impact Modular Delivery Potential to deliver to multiple cell types
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5 5 Anticipated Value Creation over the Next 12 – 15 Months Lay out what RNA editing can achieve biologically to activate pathways Present our focused pipeline of product candidates (target / indication pairs) Update on each of the assets throughout the year – starting with KRRO-121 on Jan. 27, 2026 2026 milestones Cash runway into H2 2027 Potential for partnership across our pipeline 1 1. Cash, cash equivalents and marketable securities of $102.5 million as of September 30, 2025
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6 6 Expertise in ADAR biology driving potency and translation Leveraging known mechanisms to derisk Delivery Expertise in Chemistry driving potency and drug designs Expertise in Machine Learning driving efficiency and Target ID OPERA: Our Approach for RNA Editing to GenerateProduct Candidates
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7 7 DNA Pre-mRNA mRNA Protein Edit a single A-to-I Modifying gene expression Current Focus Edit a single A-to-I • Repair a G->A mutation to correct the protein • Generate a de novo protein with preferred properties (can alter 12 amino acids) TRANSCRIPTION PROCESSING TRANSLATION RNA Editing Enables Potential for High Impact in Range of Disease Areas Human genetics guiding the possibilities
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8 Repair the Protein RNA with a missense variant TRANSLATION Pipeline Programs Primarily Focused on Generation of Protein Variants Pathogenic mutant protein Edited RNA TRANSLATION Normal “corrected” protein Example s of Repair = E34 2K AAT D, G2019S Parkinson Di sease, Dra vet’s Syndrome … Modulate Protein Function (Activate pathway) RNA TRANSLATION Normal Protein TRANSLATION Protein Variant Example sof Modulate = Hype rammo ne mia, AL S, MASH, Fibrosis… Edited RNA
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9 CONCEPT PROGRAM / INDICATION DELIVERY DISCOVERY PRECLINICAL DEVELOPMENT PHASE 1 PHASE 2 PHASE 3 Stabilize Protein KRRO-121 Hyperammonemia GalNAc (SC) Repair Pathogenic Variant AATD GalNAc (SC) Allosteric Activator Longevity (Liver) GalNAc (SC) Overcome LoF and GoF 1 Amyotrophic lateral sclerosis (ALS) Intrathecal (IT) Reg filing in 2H 2026 TDP43 Pipeline with Potential High-Value Programs and Anticipated Milestones 1De Novo protein variant to prevent toxic gain-of-function (GoF) with TDP43 aggregation, and continue downstream signaling by overcoming toxic loss-of-function (LOF) Novo Nordisk collaboration entered 12-month pause in Nov. 2025 AAT DC in 1H 2026 Terminating the REWRITE cl inical trial for KRRO-110 in AATD AMPK𝛾1 Protein variant creation Protein repair
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10 KRRO-121: Targeting Hyperammonemia
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11 Plasma Ammonia Significantly Impacts Pathology Across Multiple Diseases Novel approach to reduction of ammonia could have a profound impact to patient lives Ammonia Diet & metabolism Normal Clearance Diminished Clearance Normal excretion through kidneys ! Hyperammonemia Source: 3rd party primary market research study and analysis (April 2025); KOL interviews ▪ High ammonia leads to: ‒ Neurological impairment ‒ Frequent hospitalization ‒ Highly restricted diet ‒ Elevated infection risk ▪ Can be caused by cirrhosis or urea cycledysfunction ▪ Clinical studies have shown benefit of loweringammonia in multiple indications
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12 Our Approach: Liver-specific, GalNAc-ASO to Generate a Stable Protein Variant De novo protein variant Liver directed editing (GalNAc delivery) KRRO-121 Protein mRNA + I (G) A Edited mRNA De novo protein variant N Arg Converting a Lysine to Arginine Designed to maintain consistent ammonia clearance capacity GalNAc KRRO-121: Designed for liver-specific protein stabilization to enhance ammonia clearance capacity
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13 Urea Cycle Disorders (UCD) Hepatic Encephalopathy (HE) KRRO-121 Has Blockbuster Potential in Multiple Indications Note: 1. Severe late-onset UCD p atients; 2. Patients p rescribed rifaximin +/- lactulose with ≥1.5x normal ammonia and satisfactory liver function as assessed by laboratory values; 3. EU + U K estimate app lies U.S. epid emiology assumptions to estimated EU + UK cirrhosis population Source: 3rd party primary market research study (April 2025); KOL interviews; GlobalData; Electronic medical records analysis (d ata from 2022). All figures ap proximate. Addressable Patients 4,200 U.S. 1 5,100 EU + UK 1 80,000 U.S. 2 150,000 EU + UK3 Market Opportunity $1.5B $2B+
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14 KRRO-121: A Step Towards Activating Biological Pathways with Protein Variants ▪ Designed to stabilize an intracellular protein involved in ammonia clearance specifically in the liver ‒ GalNAc-conjugated construct to activate an existing biological pathway ‒ Creating a protein variant and learning from genetics / known biology ▪ Intended to treat patients with hyperammonemia in multiple indications ‒ UCD patients irrespective of their enzyme deficiencies in the urea cycle ‒ Patients with hepatic encephalopathy and high plasma ammonia ▪ Large unmet medical need and market opportunity in UCD and HE ▪ KRRO-121 Workshop on Jan. 27, 2026 with an anticipated regulatory filing in H2 ‘26
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1 5 15 Learnings from KRRO-110: GalNAc-conjugated AATD Asset Alpha-1 Anti-Trypsin Deficiency (AATD)
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1 6 16 Learnings from REWRITE Applied to Next Gen AATD Program ▪ RNA editing works durably in humans as demonstrated by the presence of M-AAT ▪ No dose limiting toxicities and no treatment emergent serious adverse events ▪ KRRO-110 (RNA editing oligo in a Lipid Nano Particle) did not achieve >11uM of M-AAT with single dose ▪ AATD patient serum changes the physical structure of KRRO-110 NOT seen in healthy volunteers (HV) ▪ Plasma pharmacokinetic differences seen in the components of KRRO-110 between HV and AATD patients ▪ The differences seen ex vivo are consistent with differences seen in the REWRITE trial Terminating the REWRITE clinical trial for KRRO-110 in AATD Developing a GalNAc-conjugated AATD construct KRRO-110
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1 7 17 Achieved >90% Editing of SERPINA1 Transcript using GalNAc Delivery in vivo 0 1Week SC Week 1 Results (7 days post first dose) Dosing: Q2Dx3 at 5 or 10 mg/kg '486 '810 '810 '467 0 20 40 60 80 100 27.9 69.6 73.3 91.8 66.7 84.3 % Editing 3x5 mg/kg 3x10 mg/kg NSG-PiZ MouseC57BL/6-PiZ Mouse Oligo 1 Oligo 2 Oligo 2 Oligo 3 GalNAc GalNAc-conjugated ASO RNA editing in PiZZ Mice (consistency in two models)
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1 8 18 AMPK1 Activation: Restoring Metabolic Status Activation of the Master Metabolic Regulator Designed to Improve Liver Function Potentially Restores Metabolic Signaling Without Affecting Food Intake
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1 9 19 Day 8 0 100 200 300 400 108 208 AST U/L KB356 Vehicle Day 8 0 200 400 600 800 1000 290 295 Creatine Kinase U/L KB356 Vehicle Day 8 0 100 200 300 400 122 230 ALT U/L KB356 Vehicle Lean Mice 60 U/L Lean Mice 100 U/L HepaticAMPK Activation Improved Liver Function in Obese Mice 0 2 4 6 8 95 100 105 % Body Weight Day %Body Weight 0 2 4 6 8 0 2 4 6 8 Food Consumption Day Food intake (g/day) Vehicle KB356 ***Orange arrows indicate diet refresh Normalizing Liver Function Reduction in Body Weight Despite Similar Food Intake ~20% editing was su fficient to normalize liver function and reduce body weight in mice Diet-Induced-Obese (DIO) mice were treated daily for 5 days with GalNAc-conjugated oligonucleotide at 10mg/kg and were sacrificed on day 8. GalNAc Oligo 1 Oligo 1 Oligo 1
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2 0 20 TDP43 Regulation: Restoring Function in ALS Reduced Mis-splicing Reduced Aggregates and Mis-localization of Protein Potentially Improving Cell Survival
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2 1 21 TDP-43 Variant Demonstrated Reduced Mis-splicing and Decreased Cytosolic Mis-localization in iPSC Motor Neurons TDP-43 signal quantified by confocal microscopy using thresholds defined from untreated controls, which were applied uniformly t o all images to measure TDP-43 intensity while excluding background signal Reduced Mis-splicing: Maintaining STMN2 & POLDIP3 DAPI TDP-43 Merge Cell Stressor alone Oligo 3 + Cell Stressor 0 5 10 15 20 25 Cell stressor Untreated 14 3 3 5 Mock Oligo 3 200 nM ✱✱ % Cytosolic TDP-43 Decreased Cytosolic Mis -localization of TDP -43 protein
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2 2 22 Positioned for Value Creation in 2026 and Beyond Regulatory filing for KRRO-121 anticipated in H2 2026 DC expected for GalNAc-conjugated AATD construct in H1 2026 DC expected for a 3rd GalNAc-conjugated liver asset in H2 2026 Cash runway into H2 ‘27 enabling multiple milestones1 Edit the Message Rewrite the Future 1. Cash, cash equivalents and marketable securities of $102.5 million as of September 30, 2025